4.7 Review

A perspective on oligonucleotide therapy: Approaches to patient customization

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Neurosciences

Targeted Augmentation of Nuclear Gene Output (TANGO) of Scn1a rescues parvalbumin interneuron excitability and reduces seizures in a mouse model of Dravet Syndrome

Eric R. Wengert et al.

Summary: The study demonstrates that increasing NaV1.1 levels through the TANGO mechanism can reduce seizures, prolong survival, and restore the excitability of PV interneurons in DS mice. These results support the direct therapeutic effect of TANGO on the core disease mechanism of DS.

BRAIN RESEARCH (2022)

Review Chemistry, Physical

Polyethyleneimine-Based Lipopolyplexes as Carriers in Anticancer Gene Therapies

Julia Jerzykiewicz et al.

Summary: In recent years, gene therapy for cancer treatment has gained increasing interest. Non-viral vectors, particularly lipopolyplexes consisting of nucleic acids condensed with polyvalent molecules and enclosed in lipid vesicles, show great promise as effective and safe nucleic acid carriers. This article briefly reviews the major aspects of developing such non-viral vectors based on polyethyleneimine and discusses their properties in relation to anticancer therapeutic strategies. Examples of current in vivo studies involving these lipopolyplexes and potential future developments are also presented.

MATERIALS (2022)

Article Biochemistry & Molecular Biology

Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives

Matthis Synofzik et al.

Summary: ASO therapies offer a promising disease-modifying approach for rare neurological diseases, but there is a need to focus on ultrarare or private variants to provide treatment for a larger share of patients. The emerging field of n-of-1 ASO treatment approaches requires systematic guidance and standards to allow global scaling, particularly in the European context. Genetic, regulatory, and ethical perspectives are essential for preparing and implementing n-of-1 ASO treatments to ensure both individual patient benefit and knowledge gain.

NUCLEIC ACID THERAPEUTICS (2022)

Article Clinical Neurology

Changes in brain activity with tominersen in early-manifest Huntington's disease

D. J. Hawellek et al.

Summary: The study found that patients with Huntington's disease treated with the huntingtin-lowering antisense oligonucleotide tominersen exhibited increased EEG power in the 4-8 Hz frequency range. The underlying mechanisms of these changes are unknown and may reflect neural plasticity as a consequence of the molecular pathways impacted by tominersen treatment.

BRAIN COMMUNICATIONS (2022)

Review Biotechnology & Applied Microbiology

Pharmacokinetics and Proceedings in Clinical Application of Nucleic Acid Therapeutics

Markus Herkt et al.

Summary: Oligonucleotide therapeutics are a promising class of drugs targeting RNA molecules to revolutionize disease treatment. Through chemical modifications for stability and binding, these drugs show potential in treating neurodegenerative, cardiac, and cancer disorders. With ongoing research and development, these oligonucleotide-based agents are paving the way for next-generation therapeutics.

MOLECULAR THERAPY (2021)

Review Medicine, General & Internal

Antibody-Oligonucleotide Conjugates: A Twist to Antibody-Drug Conjugates

Julien Dugal-Tessier et al.

Summary: This article summarizes the key technological advancements in the preparation of antibody-oligonucleotide conjugates (AOCs) and discusses the distinct advantages and disadvantages of AOCs as novel therapeutics. It evaluates the merits and demerits of different methods of conjugating oligonucleotides to antibodies, mainly focusing on AOC purification and analytical characterizations. Lessons learned from in vitro and in vivo studies, particularly findings related to silencing, trafficking, and cytotoxicity of the conjugates, are also summarized.

JOURNAL OF CLINICAL MEDICINE (2021)

Review Medicine, Research & Experimental

Delivery of oligonucleotide-based therapeutics: challenges and opportunities

Suzan M. Hammond et al.

Summary: The field of nucleic acid-based therapeutics has seen rapid development in recent years, with the main challenge being delivery to target tissues. The adoption of delivery technologies, such as conjugates or nanoparticles, has been a game changer for many therapeutic indications.

EMBO MOLECULAR MEDICINE (2021)

Correction Chemistry, Medicinal

The Risks of miRNA Therapeutics: In a Drug Target Perspective (vol 15, pg 721, 2021)

S. Zhang et al.

DRUG DESIGN DEVELOPMENT AND THERAPY (2021)

Review Biotechnology & Applied Microbiology

The infinite possibilities of RNA therapeutics

Evelyn C. Mollocana-Lara et al.

Summary: Although the study of RNA therapeutics was overshadowed by DNA-based therapies for many years, it is now back with force, elucidating the important role of RNA in cell regulation and disease development. Current research focuses on introducing various RNA molecules and the latest advancements in synthetic and delivery technologies, highlighting FDA-approved RNA therapies and ongoing clinical investigations.

JOURNAL OF INDUSTRIAL MICROBIOLOGY & BIOTECHNOLOGY (2021)

Letter Medicine, General & Internal

Rapid Sequencing-Based Diagnosis of Thiamine Metabolism Dysfunction Syndrome

Mallory J. Owen et al.

Summary: Speedy genetic diagnosis is crucial for infantile encephalopathy, as it can help avoid the risk of permanent neurological injury or death. A patient's genome was sequenced and diagnosed within 13 hours, leading to informed treatment.

NEW ENGLAND JOURNAL OF MEDICINE (2021)

Editorial Material Multidisciplinary Sciences

CRISPR diagnostics

Omar O. Abudayyeh et al.

SCIENCE (2021)

Review Orthopedics

The Antisense Oligonucleotide Nusinersen for Treatment of Spinal Muscular Atrophy

Amber N. Edinoff et al.

Summary: Spinal muscular atrophy (SMA) is a rare neuromuscular degenerative disease characterized by loss of spinal cord motor neurons leading to muscle wasting. Nusinersen, an ASO drug approved by FDA, has shown promising results in improving motor function and reducing ventilator requirements in SMA patients.

ORTHOPEDIC REVIEWS (2021)

Review Biochemistry & Molecular Biology

Chemistry of Peptide-Oligonucleotide Conjugates: A Review

Kristina Klabenkova et al.

Summary: This article discusses the importance of peptide-oligonucleotide conjugates in enhancing the efficiency of therapeutic nucleic acids and the related chemical synthesis methods, which are mainly divided into two strategies: stepwise solid-phase synthesis and post-synthetic conjugation.

MOLECULES (2021)

Article Oncology

An overview of precision oncology basket and umbrella trials for clinicians

Jay J. H. Park et al.

CA-A CANCER JOURNAL FOR CLINICIANS (2020)

Review Biochemistry & Molecular Biology

Aptamers Chemistry: Chemical Modifications and Conjugation Strategies

Fadwa Odeh et al.

MOLECULES (2020)

Review Pharmacology & Pharmacy

Opportunities and Challenges in the Delivery of mRNA-Based Vaccines

Abishek Wadhwa et al.

PHARMACEUTICS (2020)

Article Biochemistry & Molecular Biology

Generation and validation of structurally defined antibody-siRNA conjugates

Alex R. Nanna et al.

NUCLEIC ACIDS RESEARCH (2020)

Article Multidisciplinary Sciences

Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression

Kian Huat Lim et al.

NATURE COMMUNICATIONS (2020)

Review Medicine, Research & Experimental

Targeting RNA: A Transformative Therapeutic Strategy

Wei Yin et al.

CTS-CLINICAL AND TRANSLATIONAL SCIENCE (2019)

Article Genetics & Heredity

Mutations in ACTL6B Cause Neurodevelopmental Deficits and Epilepsy and Lead to Loss of Dendrites in Human Neurons

Scott Bell et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2019)

Review Biochemistry & Molecular Biology

Oligonucleotide therapy: An emerging focus area for drug delivery in chronic inflammatory respiratory diseases

Meenu Mehta et al.

CHEMICO-BIOLOGICAL INTERACTIONS (2019)

Review Pharmacology & Pharmacy

CRISPR/Cas System for Genome Editing: Progress and Prospects as a Therapeutic Tool

Deepak Kumar Sahel et al.

JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS (2019)

Article Genetics & Heredity

Aptamers as Therapeutic Agents: Has the Initial Euphoria Subsided?

S. K. Hassel et al.

MOLECULAR DIAGNOSIS & THERAPY (2019)

Review Chemistry, Medicinal

Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis

Veena Mathew et al.

DRUG DESIGN DEVELOPMENT AND THERAPY (2019)

Article Multidisciplinary Sciences

Specific inhibition of splicing factor activity by decoy RNA oligonucleotides

Polina Denichenko et al.

NATURE COMMUNICATIONS (2019)

Article Medicine, Research & Experimental

Natural Exon Skipping Sets the Stage for Exon Skipping as Therapy for Dystrophic Epidermolysis Bullosa

Jeroen Bremer et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2019)

Editorial Material Clinical Neurology

Oligonucleotide designed for ultimate personalized treatment

Ian Fyfe

NATURE REVIEWS NEUROLOGY (2019)

Article Medicine, General & Internal

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

J. Kim et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

Article Biochemistry & Molecular Biology

Antiviral Activity of a New Class of Chemically Modified Antisense Oligonucleotides against Influenza A Virus

A. Markov et al.

RUSSIAN JOURNAL OF BIOORGANIC CHEMISTRY (2019)

Review Clinical Neurology

Antisense oligonucleotides: the next frontier for treatment of neurological disorders

Carlo Rinaldi et al.

NATURE REVIEWS NEUROLOGY (2018)

Review Biochemistry & Molecular Biology

RD-Connect, NeurOmics and EURenOmics: collaborative European initiative for rare diseases

Hanns Lochmueller et al.

EUROPEAN JOURNAL OF HUMAN GENETICS (2018)

Review Biochemistry & Molecular Biology

Improving Small Interfering RNA Delivery In Vivo Through Lipid Conjugation

Maire F. Osborn et al.

NUCLEIC ACID THERAPEUTICS (2018)

Review Medicine, Research & Experimental

Aptamers in the Therapeutics and Diagnostics Pipelines

Harleen Kaur et al.

THERANOSTICS (2018)

Article Medicine, Research & Experimental

Liver-Targeted Anti-HBV Single-Stranded Oligonucleotides with Locked Nucleic Acid Potently Reduce HBV Gene Expression In Vivo

Hassan Javanbakht et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2018)

Article Medicine, Research & Experimental

Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models

Kalyan Dulla et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2018)

Article Clinical Neurology

Mutant superoxide dismutase aggregates from human spinal cord transmit amyotrophic lateral sclerosis

Elaheh Ekhtiari Bidhendi et al.

ACTA NEUROPATHOLOGICA (2018)

Editorial Material Medicine, General & Internal

The history and development of N-of-1 trials

R. D. Mirza et al.

JOURNAL OF THE ROYAL SOCIETY OF MEDICINE (2017)

Review Biotechnology & Applied Microbiology

The chemical evolution of oligonucleotide therapies of clinical utility

Anastasia Khvorova et al.

NATURE BIOTECHNOLOGY (2017)

Review Biotechnology & Applied Microbiology

Oligonucleotide therapies for disorders of the nervous system

Olga Khorkova et al.

NATURE BIOTECHNOLOGY (2017)

Review Chemistry, Medicinal

Eteplirsen in the treatment of Duchenne muscular dystrophy

Kenji Rowel Q. Lim et al.

DRUG DESIGN DEVELOPMENT AND THERAPY (2017)

Review Biochemistry & Molecular Biology

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

Annemieke Aartsma-Rus et al.

NUCLEIC ACID THERAPEUTICS (2017)

Article Biochemistry & Molecular Biology

Molecular Mechanisms of Antisense Oligonucleotides

Stanley T. Crooke

NUCLEIC ACID THERAPEUTICS (2017)

Review Medicine, Research & Experimental

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

Yuanyu Huang

MOLECULAR THERAPY-NUCLEIC ACIDS (2017)

Article Biochemistry & Molecular Biology

Cellular localization of long non-coding RNAs affects silencing by RNAi more than by antisense oligonucleotides

Kim A. Lennox et al.

NUCLEIC ACIDS RESEARCH (2016)

Review Pharmacology & Pharmacy

Polymeric-based particulate systems for delivery of therapeutic proteins

Muhammad Sajid Hamid Akash et al.

PHARMACEUTICAL DEVELOPMENT AND TECHNOLOGY (2016)

Article Medicine, Research & Experimental

Therapeutic correction of ApoER2 splicing in Alzheimer's disease mice using antisenseoligonucleotides

Anthony J. Hinrich et al.

EMBO MOLECULAR MEDICINE (2016)

Article Clinical Neurology

Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience

Manon Hache et al.

JOURNAL OF CHILD NEUROLOGY (2016)

Article Chemistry, Multidisciplinary

Development of antibody-siRNA conjugate targeted to cardiac and skeletal muscles

Tsukasa Sugo et al.

JOURNAL OF CONTROLLED RELEASE (2016)

Review Chemistry, Multidisciplinary

Progress in the development of lipopolyplexes as efficient non-viral gene delivery systems

Mehdi Rezaee et al.

JOURNAL OF CONTROLLED RELEASE (2016)

Review Biotechnology & Applied Microbiology

Applications of CRISPR technologies in research and beyond

Rodolphe Barrangou et al.

NATURE BIOTECHNOLOGY (2016)

Article Neurosciences

Increased 4R-Tau Induces Pathological Changes in a Human-Tau Mouse Model

Kathleen M. Schoch et al.

NEURON (2016)

Article Biotechnology & Applied Microbiology

The real cost of sequencing: scaling computation to keep pace with data generation

Paul Muir et al.

GENOME BIOLOGY (2016)

Review Pharmacology & Pharmacy

Drug delivery system of therapeutic oligonucleotides

Yutaro Asami et al.

DRUG DISCOVERIES AND THERAPEUTICS (2016)

Review Pharmacology & Pharmacy

Bioconjugates for targeted delivery of therapeutic oligonucleotides

Xin Ming et al.

ADVANCED DRUG DELIVERY REVIEWS (2015)

Review Pharmacology & Pharmacy

Delivery of oligonucleotides with lipid nanoparticles

Yuhua Wang et al.

ADVANCED DRUG DELIVERY REVIEWS (2015)

Review Pharmacology & Pharmacy

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

Richard S. Geary et al.

ADVANCED DRUG DELIVERY REVIEWS (2015)

Article Genetics & Heredity

DVL1 Frameshift Mutations Clustering in the Penultimate Exon Cause Autosomal-Dominant Robinow Syndrome

Janson White et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2015)

Article Biochemistry & Molecular Biology

Endosome Escape Strategies for Improving the Efficacy of Oligonucleotide Delivery Systems

Y. Wan et al.

CURRENT MEDICINAL CHEMISTRY (2015)

Review Immunology

The roles of CRISPR-Cas systems in adaptive immunity and beyond

Rodolphe Barrangou

CURRENT OPINION IN IMMUNOLOGY (2015)

Review Biochemistry & Molecular Biology

Aptamer Oligonucleotides: Novel Potential Therapeutic Agents in Autoimmune Disease

Weibin Li et al.

NUCLEIC ACID THERAPEUTICS (2015)

Review Medicine, Research & Experimental

siRNA Versus miRNA as Therapeutics for Gene Silencing

Jenny K. W. Lam et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2015)

Review Immunology

CpG Oligonucleotides as Cancer Vaccine Adjuvants

Hidekazu Shirota et al.

VACCINES (2015)

Article Neurosciences

Oligonucleotide-based therapy for neurodegenerative diseases

Iddo Magen et al.

BRAIN RESEARCH (2014)

Article Pharmacology & Pharmacy

Phosphorothioate Oligonucleotides: Effectiveness and Toxicity

Tommaso Iannitti et al.

CURRENT DRUG TARGETS (2014)

Review Chemistry, Multidisciplinary

PEG - A versatile conjugating ligand for drugs and drug delivery systems

Atul Kolate et al.

JOURNAL OF CONTROLLED RELEASE (2014)

Review Biochemistry & Molecular Biology

Cell-penetrating peptides: 20 years later, where do we stand?

Cherine Bechara et al.

FEBS LETTERS (2013)

Review Biochemistry & Molecular Biology

RNA-Based Therapeutics: Current Progress and Future Prospects

John C. Burnett et al.

CHEMISTRY & BIOLOGY (2012)

Review Biotechnology & Applied Microbiology

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

Ryszard Kole et al.

NATURE REVIEWS DRUG DISCOVERY (2012)

Review Medicine, Research & Experimental

Oligonucleotide therapeutic approaches for Huntington disease

Dinah W. Y. Sah et al.

JOURNAL OF CLINICAL INVESTIGATION (2011)

Review Biotechnology & Applied Microbiology

The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy

Qi-Long Lu et al.

MOLECULAR THERAPY (2011)

Review Biotechnology & Applied Microbiology

Aptamers as therapeutics

Anthony D. Keefe et al.

NATURE REVIEWS DRUG DISCOVERY (2010)

Review Biochemistry & Molecular Biology

Antisense oligonucleotide pharmacokinetics and metabolism

Richard S. Geary

EXPERT OPINION ON DRUG METABOLISM & TOXICOLOGY (2009)

Review Pharmacology & Pharmacy

Emerging oligonucleotide therapies for asthma and chronic obstructive pulmonary disease

Rosanne M. Seguin et al.

EXPERT OPINION ON INVESTIGATIONAL DRUGS (2009)

Review Biochemistry & Molecular Biology

siRNA and Innate Immunity

Marjorie Robbins et al.

OLIGONUCLEOTIDES (2009)

Article Biotechnology & Applied Microbiology

Impact of target mRNA structure on siRNA silencing efficiency: A large-scale study

Joseph A. Gredell et al.

BIOTECHNOLOGY AND BIOENGINEERING (2008)

Article Pharmacology & Pharmacy

Challenges and Opportunities for Local and Systemic Delivery of siRNA and Antisense Oligonucleotides

L. Sepp-Lorenzino et al.

CLINICAL PHARMACOLOGY & THERAPEUTICS (2008)

Review Pharmacology & Pharmacy

Antisense, RNAi, and gene silencing strategies for therapy: Mission possible or impossible?

Elizabeth R. Rayburn et al.

DRUG DISCOVERY TODAY (2008)

Review Biotechnology & Applied Microbiology

Overcoming the innate immune response to small interfering RNA

Adam Judge et al.

HUMAN GENE THERAPY (2008)

Review Biochemistry & Molecular Biology

Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides

Rudy Juliano et al.

NUCLEIC ACIDS RESEARCH (2008)

Article Chemistry, Analytical

Aptamer-based biosensors

Shiping Song et al.

TRAC-TRENDS IN ANALYTICAL CHEMISTRY (2008)

Review Medicine, Research & Experimental

Chemical modification: the key to clinical application of RNA interference?

David R. Corey

JOURNAL OF CLINICAL INVESTIGATION (2007)

Article Pharmacology & Pharmacy

Cell-penetrating peptides as vectors for peptide, protein and oligonucleotide delivery

Maarja Mae et al.

CURRENT OPINION IN PHARMACOLOGY (2006)

Article Pharmacology & Pharmacy

Antisense oligonucleotides: From design to therapeutic application

JHP Chan et al.

CLINICAL AND EXPERIMENTAL PHARMACOLOGY AND PHYSIOLOGY (2006)

Review Pharmacology & Pharmacy

Chemical modification of gene silencing oligonucleotides for drug discovery and development

XL Chen et al.

DRUG DISCOVERY TODAY (2005)

Article Pharmacology & Pharmacy

Pegaptanib - In exudative age-related macular degeneration

MAA Siddiqui et al.

Review Pharmacology & Pharmacy

Toxicology of antisense therapeutics

TLH Jason et al.

TOXICOLOGY AND APPLIED PHARMACOLOGY (2004)

Review Chemistry, Medicinal

Innovations in oligonucleotide drug delivery

MA Lysik et al.

JOURNAL OF PHARMACEUTICAL SCIENCES (2003)

Review Chemistry, Multidisciplinary

A DNA aptamer as a new target-specific chiral selector for HPLC

M Michaud et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2003)

Article Biochemistry & Molecular Biology

Cell-penetrating peptides - A reevaluation of the mechanism of cellular uptake

JP Richard et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2003)

Review Biochemistry & Molecular Biology

CpG-oligonucleotides for cancer immunotherapy: Review of the literature and potential applications in malignant glioma

AF Carpentier et al.

FRONTIERS IN BIOSCIENCE (2003)

Article Biochemistry & Molecular Biology

Lipid-mediated delivery of oligonucleotide to pulmonary endothelium

Z Ma et al.

AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY (2002)

Article Biochemistry & Molecular Biology

Design and characterization of decoy oligonucleotides containing locked nucleic acids

R Crinelli et al.

NUCLEIC ACIDS RESEARCH (2002)

Article Medicine, Research & Experimental

Therapeutic applications of transcription factor decoy oligonucleotides

MJ Mann et al.

JOURNAL OF CLINICAL INVESTIGATION (2000)