Pharmaceuticals Promoting Premature Termination Codon Readthrough: Progress in Development

Article Biotechnology & Applied Microbiology

Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis

Catherine Leroy et al.

Summary: DAP can correct nonsense mutations in genetic diseases, restore gene function, and has therapeutic potential.

MOLECULAR THERAPY (2023)

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Review Hematology

Targeting cereblon in hematologic malignancies

Ota Fuchs

Summary: The protein cereblon (CRBN) functions as a substrate receptor of the E3 ubiquitin ligase complex CRL4CRBN, mediating selective protein ubiquitination and degradation. This review discusses the development of novel drugs, such as molecular glues and PROTACs, which target CRBN and show increased efficacy in hematologic malignancies. These drugs stimulate the interaction between CRBN and disease-promoting proteins, leading to polyubiquitination and degradation of traditionally difficult-to-target proteins. The review also covers the competition between CRBN neosubstrates and endogenous proteins, as well as the pharmacology, combination therapies, and mechanisms of resistance to CRBN-based modulators or PROTACs.

BLOOD REVIEWS (2023)

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Article Biochemistry & Molecular Biology

Nonselective TRPC channel inhibition and suppression of aminoglycoside-induced premature termination codon readthrough by the small molecule AC1903

Alireza Baradaran-Heravi et al.

Summary: In this study, the researchers investigated the effects of AC1903, a selective TRPC5 inhibitor, on the cellular uptake of G418 and PTC readthrough in cancer cells and patient-derived keratinocytes. They found that AC1903 consistently suppressed G418 uptake and readthrough, indicating that nonselective cation channels, specifically TRPC channels, contribute to the variable effects of aminoglycosides. The study also revealed that AC1903 inhibits multiple TRPC channels and has previously unrecognized targets, which should be considered when interpreting experimental results.

JOURNAL OF BIOLOGICAL CHEMISTRY (2022)

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Article Respiratory System

Functional Restoration of CFTR Nonsense Mutations in Intestinal Organoids

E. de Poel et al.

Summary: This study assessed the effect of drugs targeting translational readthrough (RT), nonsense mRNA mediated decay (NMD), and CFTR protein modulation on CFTR function in intestinal organoids of people with cystic fibrosis. The results showed that a quintuple combination of drugs can effectively rescue PTC CFTR protein function, reaching clinical relevance.

JOURNAL OF CYSTIC FIBROSIS (2022)

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Review Pharmacology & Pharmacy

The potential value of amlexanox in the treatment of cancer: Molecular targets and therapeutic perspectives

Christian Bailly

Summary: Amlexanox is a drug that has been used for decades to treat mouth aphthous ulcers and is now being considered for the treatment of diabetes and obesity. It has been found to have anticancer effects in various tumor models, and its efficacy can be enhanced when combined with other drugs. The drug has multiple protein targets and its molecular basis of action is discussed in this review. The use of Amlexanox as a cancer treatment should be further explored, and the development of new analogues is encouraged.

BIOCHEMICAL PHARMACOLOGY (2022)

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Article Biochemistry & Molecular Biology

Translational Read-Through Drugs (TRIDs) Are Able to Restore Protein Expression and Ciliogenesis in Fibroblasts of Patients with Retinitis Pigmentosa Caused by a Premature Termination Codon in FAM161A

Avigail Beryozkin et al.

Summary: Ataluren and Gentamicin, as translational readthrough drugs, have been shown to effectively restore expression, localization, and function of the FAM161A protein in fibroblasts from retinitis pigmentosa patients. These drugs also restored ciliogenesis and cilia length, with Ataluren showing better results compared to Gentamicin.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2022)

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Article Neurosciences

Influence of novel readthrough agents on myelin protein zero translation in the peripheral nervous system

Yoshinori Otani et al.

Summary: Translational readthrough-inducing agents have been developed to treat nonsense mutations in hereditary diseases. This study evaluated the activities of novel negamycin-derived readthrough agents and their impact on the sciatic nerve. The results showed that these agents enhanced programmed translational readthrough and had less damaging effects on the nerve compared to traditional readthrough agents.

NEUROPHARMACOLOGY (2022)

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Article Multidisciplinary Sciences

Ataluren binds to multiple protein synthesis apparatus sites and competitively inhibits release factor-dependent termination

Shijie Huang et al.

Summary: This study identifies the binding sites of the translation readthrough inducing drug (TRID) ataluren on the ribosome, and reveals how it inhibits termination at nonsense codons.

NATURE COMMUNICATIONS (2022)

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Article Dermatology

Molecular and Clinical Outcomes After Intravenous Gentamicin Treatment for Patients With Junctional Epidermolysis Bullosa Caused by Nonsense Variants

Daniel Mosallaei et al.

Summary: This study evaluated the safety and efficacy of intravenous gentamicin readthrough therapy in JEB patients. The results showed that gentamicin treatment induced readthrough of nonsense variants, restored functional laminin 332 expression, and promoted wound healing and closure in a 3-month period.

JAMA DERMATOLOGY (2022)

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Article Dermatology

Evaluation of Systemic Gentamicin as Translational Readthrough Therapy for a Patient With Epidermolysis Bullosa Simplex With Muscular Dystrophy Owing to PLEC1 Pathogenic Nonsense Variants

Lucia Martinez-Santamaria et al.

Summary: This case report focused on a woman with EBS-MD who received gentamicin treatment for two courses, showing improvements in mucocutaneous involvement, muscle weakness, and quality of life. Laboratory tests and clinical scales showed positive outcomes, with gentamicin treatment leading to plectin expression in the skin for at least 5 months, and improvements in skeletal and respiratory muscle function.

JAMA DERMATOLOGY (2022)

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Article Biochemistry & Molecular Biology

Novel Translational Read-through-Inducing Drugs as a Therapeutic Option for Shwachman-Diamond Syndrome

Valentino Bezzerri et al.

Summary: The study evaluated the effect of a panel of ataluren analogues in treating Shwachman-Diamond syndrome (SDS). The results showed that NV848 can restore SBDS protein synthesis, improve myeloid differentiation, and reduce the number of dysplastic granulocytes, providing new possibilities for the treatment of SDS.

BIOMEDICINES (2022)

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Article Biochemistry & Molecular Biology

Generation of the First Human In Vitro Model for McArdle Disease Based on iPSC Technology

Maria del Carmen Ortuno-Costela et al.

Summary: This article describes the establishment of the first human iPSC-based model for McArdle disease and the testing of two breakthrough compounds for potential treatment. The developed model will serve as a platform for studying the pharmacological activity of drugs or compounds against this disease.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2022)

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Article Cell Biology

Translational readthrough of nonsense mutant TP53 by mRNA incorporation of 5-Fluorouridine

Mireia Palomar-Siles et al.

Summary: FUr can induce the expression of full-length p53 in tumor cells carrying R213X nonsense mutant TP53, leading to apoptosis and cell death. FUr can also restore the expression of full-length p53 in human tumor xenografts carrying TP53 R213X mutation.

CELL DEATH & DISEASE (2022)

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Article Chemistry, Medicinal

Guanidino quinazolines and pyrimidines promote readthrough of premature termination codons in cells with native nonsense mutations

Christie Morrill et al.

Summary: Using small molecules to induce readthrough is an effective method for treating genetic diseases and cancers. This study introduces a series of novel compounds that show potential for inducing readthrough in cells, either as combination therapy or standalone treatment.

BIOORGANIC & MEDICINAL CHEMISTRY LETTERS (2022)

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Article Multidisciplinary Sciences

2-Guanidino-quinazoline promotes the readthrough of nonsense mutations underlying human genetic diseases

Laure Bidou et al.

Summary: In this study, a new drug was developed and evaluated for its clinical potential in treating genetic diseases caused by premature termination codons (PTCs). The drug, TLN468, was found to be more effective than the currently used gentamicin and acted on a broader range of sequences without affecting normal stop codon readthrough.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2022)

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Article Cell & Tissue Engineering

Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-associated Leber congenital amaurosis

Amy Leung et al.

Summary: Leber congenital amaurosis type 4 (LCA4) is a severe sight impairment disease caused by AIPL1 mutations. Researchers used induced pluripotent stem cells (iPSCs) derived from renal epithelial cells to generate retinal organoids and found that the photoreceptor cells in these organoids exhibited the characteristics of LCA4. They also discovered a potential therapeutic drug, but its ability to restore the photoreceptor cells was limited.

STEM CELL REPORTS (2022)

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Article Medicine, Research & Experimental

Small-molecule eRF3a degraders rescue CFTR nonsense mutations by promoting premature termination codon readthrough

Rhianna E. Lee et al.

Summary: Cell lines created by overexpression of Bmi-1 and hTERT can faithfully mimic the response of primary airway epithelial cells to CFTR modulators in individuals with cystic fibrosis (CF). The therapeutic approach using Cereblon E3 ligase modulators targeting eukaryotic release factor 3a (eRF3a) can rescue CFTR nonsense mutations and also diminish epithelial sodium channel (ENaC) function.

JOURNAL OF CLINICAL INVESTIGATION (2022)

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Article Biology

Deciphering the molecular mechanism of stop codon readthrough

Martine Palma et al.

Summary: Recognition of the stop codon is crucial for terminating translation and synthesizing the correct size protein. Stop codon readthrough can occur under specific conditions, leading to different protein isoforms, and has potential therapeutic implications for genetic diseases caused by nonsense mutations.

BIOLOGICAL REVIEWS (2021)

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Review Chemistry, Medicinal

Ataluren-Promising Therapeutic Premature Termination Codon Readthrough Frontrunner

Sylwia Michorowska

Summary: Around 12% of disease-causing mutations are in-frame nonsense mutations, leading to truncated proteins with little or no function. Ataluren is the only approved readthrough agent for treating patients with nonsense mutation Duchenne muscular dystrophy. Its journey from identification to clinical trials, pharmacokinetics, and potential applications in treating other diseases with underlying nonsense mutations are summarized.

PHARMACEUTICALS (2021)

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Article Pharmacology & Pharmacy

Phase 1 Renal Impairment Trial Results Supports Targeted Individualized Dosing of ELX-02 in Patients With Nephropathic Cystinosis

Thomas Haverty et al.

Summary: This study aimed to evaluate the pharmacokinetics and safety of ELX-02 in patients with renal impairment, developing an eGFR-PK model for individualized dosing in nephropathic cystinosis patients. ELX-02 exhibited good tolerability in both renal impairment and nephropathic cystinosis populations, with reduced clearance, increased exposure, and prolonged renal elimination proportional to decreases in eGFR.

JOURNAL OF CLINICAL PHARMACOLOGY (2021)

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Article Multidisciplinary Sciences

Ataluren and aminoglycosides stimulate read-through of nonsense codons by orthogonal mechanisms

Martin Y. Ng et al.

Summary: This study investigates the effects of ataluren and G418 on read-through, finding that ataluren exclusively stimulates read-through by inhibiting release factor activity, while G418 increases near-cognate tRNA mispairing with a PSC. The low toxicity of ataluren suggests that developing new TRIDs aimed at inhibiting termination should be a priority in combating PSC diseases. Additionally, the research provides rate measurements of certain steps during the eukaryotic translation elongation cycle, showing how these rates are altered when cognate tRNA is replaced by near-cognate tRNA +/- TRIDs.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2021)

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Article Pharmacology & Pharmacy

A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Escalation Study to Evaluate the Safety and Pharmacokinetics of ELX-02 in Healthy Subjects

Andi Leubitz et al.

Summary: ELX-02 is being developed as a therapy for genetic diseases caused by nonsense mutations. The phase 1 trial in healthy volunteers showed dose-proportional plasma exposure with no accumulation, but some mild to moderate injection site reactions. Although some subjects experienced changes in auditory and hearing thresholds, these resolved or improved after discontinuation of the drug. No severe adverse events were reported, supporting further evaluation in phase 2 trials.

CLINICAL PHARMACOLOGY IN DRUG DEVELOPMENT (2021)

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Letter Ophthalmology

Topical gentamicin ointment induces LAMB3 nonsense mutation readthrough and improves corneal erosions in a patient with junctional epidermolysis bullosa

Jia-Horung Hung et al.

CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY (2021)

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Article Respiratory System

Targeting G542X CFTR nonsense alleles with ELX-02 restores CFTR function in human-derived intestinal organoids

Daniel K. Crawford et al.

Summary: ELX-02 promotes read-through of mRNA transcripts with nonsense mutations in CF patient-derived organoids, leading to increased CFTR activity and expression. These findings support the potential of ELX-02 as a read-through agent for CF caused by the G542X allele.

JOURNAL OF CYSTIC FIBROSIS (2021)

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Article Chemistry, Medicinal

CC-90009: A Cereblon E3 Ligase Modulating Drug That Promotes Selective Degradation of GSPT1 for the Treatment of Acute Myeloid Leukemia

Joshua D. Hansen et al.

Summary: Acute myeloid leukemia poses a significant clinical challenge with poor survival and high relapse rates. CC-90009, a novel protein degrader targeting GSPT1 for proteasomal degradation, represents a promising therapeutic approach currently in phase 1 clinical development.

JOURNAL OF MEDICINAL CHEMISTRY (2021)

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Review Pediatrics

Application of topical gentamicin-a new era in the treatment of genodermatosis

Shan Wang et al.

Summary: Studies have shown that topical application of gentamicin has yielded promising results in the treatment of genetic skin diseases, including Nagashima-type palmoplantar keratosis, epidermolysis bullosa, Hailey-Hailey disease, and hereditary hypotrichosis simplex of the scalp.

WORLD JOURNAL OF PEDIATRICS (2021)

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Article Multidisciplinary Sciences

Cellular variability of nonsense-mediated mRNA decay

Hanae Sato et al.

Summary: The author developed a single-cell reporter system to investigate cell-to-cell variability of NMD efficiency. The study revealed a wide range of NMD efficiency in different cells, potentially linked to the expression levels of surveillance factors.

NATURE COMMUNICATIONS (2021)

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Article Clinical Neurology

Ataluren for drug-resistant epilepsy in nonsense variant-mediated Dravet syndrome and CDKL5 deficiency disorder

Orrin Devinsky et al.

Summary: This study investigated the safety and efficacy of ataluren in children with DS and CDD caused by nonsense variants, finding that ataluren was not effective in reducing seizure frequency or improving cognitive, motor, behavioral function, or quality of life.

ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY (2021)

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Review Biochemistry & Molecular Biology

Nonsense suppression therapies in human genetic diseases

Patricia Martins-Dias et al.

Summary: Nonsense mutations can lead to dysfunctional proteins, but nonsense suppression therapy has the potential to restore protein function and treat a variety of genetic disorders. However, the efficiency of suppression may be influenced by nonsense-mediated decay, highlighting the importance of using NMD inhibitors or readthrough-compound potentiators to enhance therapeutic effects.

CELLULAR AND MOLECULAR LIFE SCIENCES (2021)

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Article Health Care Sciences & Services

Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients

Craig M. McDonald et al.

Summary: The study found that ataluren plus standard of care can delay the age at loss of ambulation and the decline in respiratory function for patients with nonsense mutation Duchenne muscular dystrophy, providing benefits for both ambulatory and non-ambulatory patients compared to standard of care alone.

JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH (2021)

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Article Biochemistry & Molecular Biology

Properties of Non-Aminoglycoside Compounds Used to Stimulate Translational Readthrough of PTC Mutations in Primary Ciliary Dyskinesia

Maciej Dabrowski et al.

Summary: The study identified several non-aminoglycoside compounds with potential to induce PTC-readthrough, which demonstrated minimal negative impact on cell viability and function compared to aminoglycosides, although with lower efficiency.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)

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Article Biochemistry & Molecular Biology

Effect of small molecule eRF3 degraders on premature termination codon readthrough

Alireza Baradaran-Heravi et al.

Summary: The study demonstrates that PTC readthrough by aminoglycoside G418 is significantly enhanced by eRF3a and eRF3b siRNAs, as well as cereblon E3 ligase modulators CC-885 and CC-90009. Degradation of eRF3 reduces eRF1 levels, upregulates UPF1, and selectively stabilizes TP53 transcripts carrying nonsense mutations, indicating NMD suppression. CC-90009, in particular, shows less toxicity compared to CC-885 and enhances PTC readthrough when combined with aminoglycosides in various genetic diseases with nonsense mutations.

NUCLEIC ACIDS RESEARCH (2021)

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Article Biochemistry & Molecular Biology

Small molecule Y-320 stimulates ribosome biogenesis, protein synthesis, and aminoglycoside-induced premature termination codon readthrough

Sara Hosseini-Farahabadi et al.

Summary: The research identified a small molecule Y-320 that enhances premature termination codon (PTC) readthrough induced by G418, increasing ribosome biogenesis and protein synthesis. Y-320 enhances PTC readthrough through autocrine chemokine signaling pathway, supporting a model where increased protein synthesis enables enhanced PTC readthrough.

PLOS BIOLOGY (2021)

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Article Multidisciplinary Sciences

A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion

Jyoti Sharma et al.

Summary: In this study, compounds with readthrough activity were identified and shown to reduce premature termination associated with cystic fibrosis by lowering eRF1 levels. These compounds, including SRI-41315 and SRI-37240, have potential as promising treatment strategies for diseases caused by PTCs.

NATURE COMMUNICATIONS (2021)

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Article Biochemistry & Molecular Biology

Stop Codon Context-Specific Induction of Translational Readthrough

Mirco Schilff et al.

Summary: The study explores the impact of stop codon context on the treatment strategy for genetic premature termination codon diseases and finds that SCC plays a significant role in readthrough stimulation and drug concentration selection.

BIOMOLECULES (2021)

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Article Dermatology

Effect of Gentamicin Ointment in Patients with Nagashima-type Palmoplantar Keratosis: A Double-blind Vehicle-controlled Study

Yue Li et al.

Summary: Gentamicin ointment showed positive responses in treating Nagashima-type palmoplantar keratosis caused by nonsense mutations, with significant improvements in symptoms of hyperkeratosis and foul smell compared to vehicle ointment, although there was no difference in effect on erythema.

ACTA DERMATO-VENEREOLOGICA (2021)

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Article Biochemistry & Molecular Biology

Effect of small molecule eRF3 degraders on premature termination codon readthrough

Alireza Baradaran-Heravi et al.

Summary: Premature termination codon (PTC) readthrough is a potential treatment for genetic diseases caused by nonsense mutations. Aminoglycosides induce low levels of PTC readthrough but are toxic. Finding compounds to enhance PTC readthrough by aminoglycosides or reduce toxicity remains a challenge.

NUCLEIC ACIDS RESEARCH (2021)

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Article Hematology

Variable readthrough responsiveness of nonsense mutations in hemophilia A

Lluis Martorell et al.

HAEMATOLOGICA (2020)

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Article Dermatology

Treatment of hereditary hypotrichosis simplex of the scalp with topical gentamicin

A. Peled et al.

BRITISH JOURNAL OF DERMATOLOGY (2020)

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Article Biochemistry & Molecular Biology

Frontotemporal dementia non-sense mutation of progranulin rescued by aminoglycosides

Lisha Kuang et al.

HUMAN MOLECULAR GENETICS (2020)

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Article Respiratory System

Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF)

M. W. Konstan et al.

JOURNAL OF CYSTIC FIBROSIS (2020)

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Letter Dermatology

Gentamicin induces COL17A1 nonsense mutation readthrough in junctional epidermolysis bullosa

Yue Li et al.

JOURNAL OF DERMATOLOGY (2020)

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Article Chemistry, Medicinal

Pharmacophore-Based Design of New Chemical Scaffolds as Translational Readthrough-Inducing Drugs (TRIDs)

Marco Tutone et al.

ACS MEDICINAL CHEMISTRY LETTERS (2020)

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Article Biology

Stop codon context influences genome-wide stimulation of termination codon readthrough by aminoglycosides

Jamie R. Wangen et al.

ELIFE (2020)

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Article Health Care Sciences & Services

Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

Eugenio Mercuri et al.

JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH (2020)

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Article Pharmacology & Pharmacy

ELX-02 Generates Protein via Premature Stop Codon Read-Through without Inducing Native Stop Codon Read-Through Proteins

Daniel K. Crawford et al.

JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS (2020)

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Article Multidisciplinary Sciences

2,6-Diaminopurine as a highly potent corrector of UGA nonsense mutations

Carole Trzaska et al.

NATURE COMMUNICATIONS (2020)

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Article Biotechnology & Applied Microbiology

Gentamicin Induces Laminin 332 and Improves Wound Healing in Junctional Epidermolysis Bullosa Patients with Nonsense Mutations

Andrew Kwong et al.

MOLECULAR THERAPY (2020)

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Review Biochemistry & Molecular Biology

Suppression of Nonsense Mutations by New Emerging Technologies

Pedro Morais et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)

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Article Pharmacology & Pharmacy

ELX-02: an investigational read-through agent for the treatment of nonsense mutation-related genetic disease

Eitan Kerem

EXPERT OPINION ON INVESTIGATIONAL DRUGS (2020)

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Article Hematology

CC-90009, a novel cereblon E3 ligase modulator targets acute myeloid leukemia blasts and leukemia stem cells

Christine Surka et al.

BLOOD (2020)

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Review Biochemistry & Molecular Biology

Molecular Insights into Determinants of Translational Readthrough and Implications for Nonsense Suppression Approaches

Silvia Lombardi et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)

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Article Biochemistry & Molecular Biology

Translational Read-Through Therapy of RPGR Nonsense Mutations

Christine Voessing et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)

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Article Medicine, Research & Experimental

RNA-based therapies in animal models of Leber congenital amaurosis causing blindness

Xia Wang et al.

PRECISION CLINICAL MEDICINE (2020)

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Article Immunology

Circulating Truncated Alpha-1 Antitrypsin Glycoprotein in Patient Plasma Retains Anti-Inflammatory Capacity

Emer P. Reeves et al.

JOURNAL OF IMMUNOLOGY (2019)

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Article Chemistry, Medicinal

Deciphering the Nonsense Readthrough Mechanism of Action of Ataluren: An in Silico Compared Study

Marco Tutone et al.

ACS MEDICINAL CHEMISTRY LETTERS (2019)

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Article Dermatology

Attenuation of Severe Generalized Junctional Epidermolysis Bullosa by Systemic Treatment with Gentamicin

Johanna Hammersen et al.

DERMATOLOGY (2019)

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Article Chemistry, Medicinal

2-Aminothiazole-4-carboxamides Enhance Readthrough of Premature Termination Codons by Aminoglycosides

Safwat M. Rabea et al.

ACS MEDICINAL CHEMISTRY LETTERS (2019)

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Article Biochemistry & Molecular Biology

Targeting Translation Termination Machinery with Antisense Oligonucleotides for Diseases Caused by Nonsense Mutations

Lulu Huang et al.

NUCLEIC ACID THERAPEUTICS (2019)

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Review Biochemistry & Molecular Biology

Strategies against Nonsense: Oxadiazoles as Translational Readthrough-Inducing Drugs (TRIDs)

Ambra Campofelice et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2019)

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Article Multidisciplinary Sciences

The antimalarial drug mefloquine enhances TP53 premature termination codon readthrough by aminoglycoside G418

Michael W. Ferguson et al.

PLOS ONE (2019)

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Article Biochemistry & Molecular Biology

Aminoglycoside drugs induce efficient read-through of CDKL5 nonsense mutations, slightly restoring its kinase activity

Maria Fazzari et al.

RNA BIOLOGY (2019)

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Article Chemistry, Medicinal

New Negamycin-Based Potent Readthrough Derivative Effective against TGA-Type Nonsense Mutations

Keisuke Hamada et al.

ACS MEDICINAL CHEMISTRY LETTERS (2019)

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Review Chemistry, Medicinal

Escin: a review of its anti-edematous, anti-inflammatory, and venotonic properties

Luca Gallelli

DRUG DESIGN DEVELOPMENT AND THERAPY (2019)

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Article Health Care Sciences & Services

Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry

Francesco Muntoni et al.

JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH (2019)

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Article Biochemistry & Molecular Biology

Ataluren for the Treatment of Usher Syndrome 2A Caused by Nonsense Mutations

Ananya Samanta et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2019)

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Article Pediatrics

The aminoglycoside geneticin permits translational readthrough of the CTNS W138X nonsense mutation in fibroblasts from patients with nephropathic cystinosis

Emma J. Brasell et al.

PEDIATRIC NEPHROLOGY (2019)

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Article Multidisciplinary Sciences

The novel aminoglycoside, ELX-02, permits CTNSW138X translational read-through and restores lysosomal cystine efflux in cystinosis

Emma J. Brasell et al.

PLOS ONE (2019)

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Article Pharmacology & Pharmacy

Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX-02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers

Andi Leubitz et al.

CLINICAL PHARMACOLOGY IN DRUG DEVELOPMENT (2019)

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Review Biotechnology & Applied Microbiology

Chemotherapeutics overcoming nonsense mutation-associated genetic diseases: medicinal chemistry of negamycin

Akihiro Taguchi et al.

JOURNAL OF ANTIBIOTICS (2018)

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Article Biochemistry & Molecular Biology

Amlexanox provides a potential therapy for nonsense mutations in the lysosomal storage disorder Aspartylglucosaminuria

Antje Banning et al.

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2018)

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Article Dermatology

Gentamicin-Induced Readthrough and Nonsense-Mediated mRNA Decay of SERPINB7 Nonsense Mutant Transcripts

Yuka Ohguchi et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2018)

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Review Biochemistry & Molecular Biology

Advances in therapeutic use of a drug-stimulated translational readthrough of premature termination codons

Maciej Dabrowski et al.

MOLECULAR MEDICINE (2018)

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Article Multidisciplinary Sciences

The effect of PTC124 on choroideremia fibroblasts and iPSC-derived RPE raises considerations for therapy

Simona Torriano et al.

SCIENTIFIC REPORTS (2018)

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Article Chemistry, Medicinal

Rescuing the CFTR protein function: Introducing 1,3,4-oxadiazoles as translational readthrough inducing drugs

Ivana Pibiri et al.

EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY (2018)

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Article Multidisciplinary Sciences

The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential

Westley J. Friesen et al.

PLOS ONE (2018)

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Article Chemistry, Medicinal

New in Vitro Assay Measuring Direct Interaction of Nonsense Suppressors with the Eukaryotic Protein Synthesis Machinery

Martin Y. Ng et al.

ACS MEDICINAL CHEMISTRY LETTERS (2018)

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Article Medicine, Research & Experimental

Gentamicin induces functional type VII collagen in recessive dystrophic epidermolysis bullosa patients

David T. Woodley et al.

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Amlexanox Enhances Premature Termination Codon Read-Through in COL7A1 and Expression of Full Length Type VII Collagen: Potential Therapy for Recessive Dystrophic Epidermolysis Bullosa

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Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

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LANCET (2017)

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Optimized approach for the identification of highly efficient correctors of nonsense mutations in human diseases

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PLOS ONE (2017)

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Aminoglycoside interactions and impacts on the eukaryotic ribosome

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PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2017)

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Characterization of new-generation aminoglycoside promoting premature termination codon readthrough in cancer cells

Laure Bidou et al.

RNA BIOLOGY (2017)

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The nucleoside analog clitocine is a potent and efficacious readthrough agent

Westley J. Friesen et al.

RNA (2017)

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Rescue of the MERTK phagocytic defect in a human iPSC disease model using translational read-through inducing drugs

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SCIENTIFIC REPORTS (2017)

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Aminoglycosides, but not PTC124 (Ataluren), rescue nonsense mutations in the leptin receptor and in luciferase reporter genes

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SCIENTIFIC REPORTS (2017)

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Article Cardiac & Cardiovascular Systems

Modeling Treatment Response for Lamin A/C Related Dilated Cardiomyopathy in Human Induced Pluripotent Stem Cells

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JOURNAL OF THE AMERICAN HEART ASSOCIATION (2017)

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2-Guanidino-quinazolines as a novel class of translation inhibitors

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BIOCHIMIE (2017)

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A flow cytometry-based reporter assay identifies macrolide antibiotics as nonsense mutation read-through agents

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JOURNAL OF MOLECULAR MEDICINE-JMM (2016)

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Discovery of Clinically Approved Agents That Promote Suppression of Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations

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AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2016)

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Clitocine potentiates TRAIL-mediated apoptosis in human colon cancer cells by promoting Mcl-1 degradation

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APOPTOSIS (2016)

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Targeting Nonsense Mutations in Diseases with Translational Read-Through-Inducing Drugs (TRIDs)

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Readthrough-Promoting Drugs Gentamicin and PTC124 Fail to Rescue Nav1.5 Function of Human-Induced Pluripotent Stem Cell-Derived Cardiomyocytes Carrying Nonsense Mutations in the Sodium Channel Gene SCN5A

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CIRCULATION-ARRHYTHMIA AND ELECTROPHYSIOLOGY (2016)

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Exploring the readthrough of nonsense mutations by non-acidic Ataluren analogues selected by ligand-based virtual screening

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EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY (2016)

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Functional rescue of REP1 following treatment with PTC124 and novel derivative PTC-414 in human choroideremia fibroblasts and the nonsense-mediated zebrafish model

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HUMAN MOLECULAR GENETICS (2016)

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Limited premature termination codon suppression by read-through agents in cystic fibrosis intestinal organoids

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JOURNAL OF CYSTIC FIBROSIS (2016)

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Novel small molecules potentiate premature termination codon readthrough by aminoglycosides

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NUCLEIC ACIDS RESEARCH (2016)

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Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression

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PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2016)

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Design of Novel Aminoglycoside Derivatives with Enhanced Suppression of Diseases-Causing Nonsense Mutations

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ACS MEDICINAL CHEMISTRY LETTERS (2016)

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COLD SPRING HARBOR PERSPECTIVES IN MEDICINE (2016)

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Enhancement of premature stop codon readthrough in the CFTR gene by Ataluren (PTC124) derivatives

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EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY (2015)

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Defects in tRNA Anticodon Loop 2′-O-Methylation Are Implicated in Nonsyndromic X-Linked Intellectual Disability due to Mutations in FTSJ1

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HUMAN MUTATION (2015)

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Nonsense suppression by near-cognate tRNAs employs alternative base pairing at codon positions 1 and 3

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PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2015)

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Translational read-through of the RP2 Arg120stop mutation in patient iPSC-derived retinal pigment epithelium cells

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HUMAN MOLECULAR GENETICS (2015)

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Evaluation of Aminoglycoside and Non-Aminoglycoside Compounds for Stop-Codon Readthrough Therapy in Four Lysosomal Storage Diseases

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Review Genetics & Heredity

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ANNUAL REVIEW OF GENOMICS AND HUMAN GENETICS, VOL 15 (2014)

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Clitocine induces apoptosis and enhances the lethality of ABT-737 in human colon cancer cells by disrupting the interaction of Mcl-1 and Bak

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CANCER LETTERS (2014)

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Discovery of Natural Products Possessing Selective Eukaryotic Readthrough Activity: 3-epi-Deoxynegamycin and Its Leucine Adduct

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Ataluren: First Global Approval

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Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway

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HUMAN MOLECULAR GENETICS (2014)

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Designer Aminoglycosides That Selectively Inhibit Cytoplasmic Rather than Mitochondrial Ribosomes Show Decreased Ototoxicity A STRATEGY FOR THE TREATMENT OF GENETIC DISEASES

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Long-term nonsense suppression therapy moderates MPS I-H disease progression

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MOLECULAR GENETICS AND METABOLISM (2014)

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Nonsense-mediated mRNA decay: Inter-individual variability and human disease

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Editorial Material Biotechnology & Applied Microbiology

Generating SM(a)RTer Compounds for Translation Termination Suppression in A-T and Other Genetic Disorders

Martin F. Lavin

MOLECULAR THERAPY (2013)

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A New Series of Small Molecular Weight Compounds Induce Read Through of All Three Types of Nonsense Mutations in the ATM Gene

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HEXEvent: a database of Human EXon splicing Events

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NUCLEIC ACIDS RESEARCH (2013)

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A Lack of Premature Termination Codon Read-Through Efficacy of PTC124 (Ataluren) in a Diverse Array of Reporter Assays

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SMRT compounds correct nonsense mutations in primary immunodeficiency and other genetic models

Richard A. Gatti

YEAR IN HUMAN AND MEDICAL GENETICS: INBORN ERRORS OF IMMUNITY III (2012)

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Readthrough of long-QT syndrome type 1 nonsense mutations rescues function but alters the biophysical properties of the channel

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A comparative evaluation of NB30, NB54 and PTC124 in translational read-through efficacy for treatment of an USH1C nonsense mutation

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Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy

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HUMAN MOLECULAR GENETICS (2012)

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The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse

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Rescue of nonsense mutations by amlexanox in human cells

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ORPHANET JOURNAL OF RARE DISEASES (2012)

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Pharmaceutical therapies to recode nonsense mutations in inherited diseases

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Sense from nonsense: therapies for premature stop codon diseases

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Statistical Analysis of Readthrough Levels for Nonsense Mutations in Mammalian Cells Reveals a Major Determinant of Response to Gentamicin

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Synthesis and evaluation of compounds that induce readthrough of premature termination codons

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BIOORGANIC & MEDICINAL CHEMISTRY LETTERS (2011)

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Readthrough of nonsense mutations in Rett syndrome: evaluation of novel aminoglycosides and generation of a new mouse model

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Suppression of CFTR premature termination codons and rescue of CFTR protein and function by the synthetic aminoglycoside NB54

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Repairing faulty genes by aminoglycosides: Development of new derivatives of geneticin (G418) with enhanced suppression of diseases-causing nonsense mutations

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Restoration of APC gene function in colorectal cancer cells by aminoglycoside- and macrolide-induced read-through of premature termination codons

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Transdermal delivery of a readthrough-inducing drug: a new approach of gentamicin administration for the treatment of nonsense mutation-mediated disorders

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Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124

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PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2010)

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Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA

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BMC NEUROSCIENCE (2009)

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Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model

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HUMAN MOLECULAR GENETICS (2009)

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Nonaminoglycoside compounds induce readthrough of nonsense mutations

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Development of Novel Aminoglycoside (NB54) with Reduced Toxicity and Enhanced Suppression of Disease-Causing Premature Stop Mutations

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Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression

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PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)

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A meta-analysis of nonsense mutations causing human genetic disease

Matthew Mort et al.

HUMAN MUTATION (2008)

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Negamycin binds to the wall of the nascent chain exit tunnel of the 50S ribosomal subunit

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ANTIMICROBIAL AGENTS AND CHEMOTHERAPY (2007)

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In vitro and ex vivo suppression by aminoglycosides of PCDH15 nonsense mutations underlying type 1 Usher syndrome

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Regulation of p53 expression in response to 5-fluorouracil in human cancer RKO cells

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CLINICAL CANCER RESEARCH (2007)

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PTC124 targets genetic disorders caused by nonsense mutations

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NATURE (2007)

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In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis:: a pilot study

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Article Genetics & Heredity

Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts

Virginia B. Mattis et al.

HUMAN GENETICS (2006)

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Letter Dermatology

Aminoglycosides as potential pharmacogenetic agents in the treatment of Hailey-Hailey disease

Richard Kellermayer et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2006)

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Aminoglycoside suppression of nonsense mutations in severe hemophilia

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BLOOD (2005)

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Effects of a number of classes of 50S inhibitors on stop codon readthrough during protein synthesis

J Thompson et al.

ANTIMICROBIAL AGENTS AND CHEMOTHERAPY (2004)

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Negamycin restores dystrophin expression in skeletal and cardiac muscles of mdx mice

M Arakawa et al.

JOURNAL OF BIOCHEMISTRY (2003)

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Gentamicin-induced correction of CFTR function in patients with cystic fibrosis and CFTR stop mutations

M Wilschanski et al.

NEW ENGLAND JOURNAL OF MEDICINE (2003)

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Pyranmycins, a novel class of aminoglycosides with improved acid stability: The SAR of D-pyranoses on ring III of pyranmycin

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ORGANIC LETTERS (2002)

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Aminoglycoside suppression of a premature stop mutation in a Cftr-/- mouse carrying a human CFTR-G542X transgene

M Du et al.

JOURNAL OF MOLECULAR MEDICINE-JMM (2002)

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Structural origins of aminoglycoside specificity for prokaryotic ribosomes

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JOURNAL OF MOLECULAR BIOLOGY (2001)

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Review Oncology

5-Fluorouracil: forty-plus and still ticking. A review of its preclinical and clinical development

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INVESTIGATIONAL NEW DRUGS (2000)

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