Related references
Note: Only part of the references are listed.A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment
Olga Mitelman et al.
JOURNAL OF NEUROMUSCULAR DISEASES (2022)
After Patient Death, FDA Places Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Trial
Alex Philippidis
HUMAN GENE THERAPY (2022)
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access
Francesca Fumagalli et al.
LANCET (2022)
Restoration of dystrophin expression in mice by suppressing a nonsense mutation through the incorporation of unnatural amino acids
Ningning Shi et al.
NATURE BIOMEDICAL ENGINEERING (2022)
Full-Length Dystrophin Restoration via Targeted Exon Addition in DMD-Patient Specific iPSCs and Cardiomyocytes
Rou Xiao et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2022)
Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors
Marta Garcia et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2022)
Large-scale genome editing based on high-capacity adenovectors and CRISPR-Cas9 nucleases rescues full-length dystrophin synthesis in DMD muscle cells
Francesca Tasca et al.
NUCLEIC ACIDS RESEARCH (2022)
IGNITE DMD phase I/II study of SGT-001 microdystrophin gene therapy for DMD: Long-term outcomes and expression update
R. Donisa Dreghici et al.
NEUROMUSCULAR DISORDERS (2022)
Generation of CD16A gene knockout human embryonic stem cell line using CRISPR/Cas9
Shunjie Wu et al.
STEM CELL RESEARCH (2022)
Delivering the CRISPR/Cas9 system for engineering gene therapies: Recent cargo and delivery approaches for clinical translation
Ruth A. Foley et al.
FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY (2022)
Gene Therapy for Duchenne Muscular Dystrophy
Nertiyan Elangkovan et al.
Journal of Neuromuscular Diseases (2021)
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial
Craig M. McDonald et al.
JOURNAL OF NEUROMUSCULAR DISEASES (2021)
Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies
Omar Sheikh et al.
EXPERT OPINION ON INVESTIGATIONAL DRUGS (2021)
Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset
Nancy J. Newman et al.
OPHTHALMOLOGY (2021)
Therapeutic Approaches for Duchenne Muscular Dystrophy: Old and New
Samuel J. Mackenzie et al.
SEMINARS IN PEDIATRIC NEUROLOGY (2021)
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial
John W. Day et al.
LANCET NEUROLOGY (2021)
Double MS2 guided restoration of genetic code in amber (TAG), opal (TGA) and ochre (TAA) stop codon
Sonali Bhakta et al.
ENZYME AND MICROBIAL TECHNOLOGY (2021)
Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial
Kathryn R. Wagner et al.
MUSCLE & NERVE (2021)
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
Julian D. Gillmore et al.
NEW ENGLAND JOURNAL OF MEDICINE (2021)
Exon skipping induced by nonsense/frameshift mutations in DMD gene results in Becker muscular dystrophy
Mariko Okubo et al.
HUMAN GENETICS (2020)
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
Diane E. Frank et al.
NEUROLOGY (2020)
Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping A Phase 2 Randomized Clinical Trial
Paula R. Clemens et al.
JAMA NEUROLOGY (2020)
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy A Nonrandomized Controlled Trial
Jerry R. Mendell et al.
JAMA NEUROLOGY (2020)
Development of a Single Construct System for Site-Directed RNA Editing Using MS2-ADAR
Tetsuto Tohama et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)
RNA editing of BFP, a point mutant of GFP, using artificial APOBEC1 deaminase to restore the genetic code
Sonali Bhakta et al.
SCIENTIFIC REPORTS (2020)
Meganuclease-Based Artificial Transcription Factors
Shingo Suzuki et al.
ACS SYNTHETIC BIOLOGY (2020)
Selective tissue targeting of synthetic nucleic acid drugs
Punit P. Seth et al.
JOURNAL OF CLINICAL INVESTIGATION (2019)
In vivo RNA editing of point mutations via RNA-guided adenosine deaminases
Dhruva Katrekar et al.
NATURE METHODS (2019)
Exons 45-55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene
Yusuke Echigoya et al.
MOLECULAR THERAPY (2019)
CRISPR Correction of Duchenne Muscular Dystrophy
Yi-Li Min et al.
ANNUAL REVIEW OF MEDICINE, VOL 70 (2019)
Comparative Activity of Adenosine Deaminase Acting on RNA (ADARs) Isoforms for Correction of Genetic Code in Gene Therapy
Md Thoufic A. Azad et al.
CURRENT GENE THERAPY (2019)
Homologous recombination and the repair of DNA double-strand breaks
William Douglass Wright et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2018)
Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
Dongsheng Duan
MOLECULAR THERAPY (2018)
Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy
Seuk-Min Ryu et al.
NATURE BIOTECHNOLOGY (2018)
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
D. Adams et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
M. D. Benson et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
E. Mercuri et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
Medical relevance of protein-truncating variants across 337,205 individuals in the UK Biobank study
Christopher DeBoever et al.
NATURE COMMUNICATIONS (2018)
Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing
Chengzu Long et al.
SCIENCE ADVANCES (2018)
Gene therapy for neurological disorders: progress and prospects
Benjamin E. Deverman et al.
NATURE REVIEWS DRUG DISCOVERY (2018)
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Craig M. McDonald et al.
LANCET (2017)
Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy
Yusuke Echigoya et al.
MOLECULAR THERAPY (2017)
Non-homologous DNA end joining and alternative pathways to double-strand break repair
Howard H. Y. Chang et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2017)
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
Florian Eichler et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model
Karima Relizani et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2017)
In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Mohammadsharif Tabebordbar et al.
SCIENCE (2016)
A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells
Courtney S. Young et al.
CELL STEM CELL (2016)
Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells
Ignazio Maggio et al.
SCIENTIFIC REPORTS (2016)
Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy
Joe W. McGreevy et al.
DISEASE MODELS & MECHANISMS (2015)
Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene Therapy
Michael E. Nance et al.
HUMAN GENE THERAPY (2015)
The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations
Catherine L. Bladen et al.
HUMAN MUTATION (2015)
Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases
David G. Ousterout et al.
MOLECULAR THERAPY (2015)
Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
David G. Ousterout et al.
NATURE COMMUNICATIONS (2015)
Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9
Hongmei Lisa Li et al.
STEM CELL REPORTS (2015)
Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases
Seung Woo Cho et al.
GENOME RESEARCH (2014)
ATALUREN TREATMENT OF PATIENTS WITH NONSENSE MUTATION DYSTROPHINOPATHY
Katharine Bushby et al.
MUSCLE & NERVE (2014)
Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV
Pablo Tebas et al.
NEW ENGLAND JOURNAL OF MEDICINE (2014)
Gene Correction of a Duchenne Muscular Dystrophy Mutation by Meganuclease-Enhanced Exon Knock-In
Linda Popplewell et al.
HUMAN GENE THERAPY (2013)
DNA targeting specificity of RNA-guided Cas9 nucleases
Patrick D. Hsu et al.
NATURE BIOTECHNOLOGY (2013)
High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells
Yanfang Fu et al.
NATURE BIOTECHNOLOGY (2013)
Repair of Strand Breaks by Homologous Recombination
Maria Jasin et al.
COLD SPRING HARBOR PERSPECTIVES IN BIOLOGY (2013)
Endonucleases: tools to correct the dystrophin gene
Joel Rousseau et al.
JOURNAL OF GENE MEDICINE (2011)
Read-Through Strategies for Suppression of Nonsense Mutations in Duchenne/Becker Muscular Dystrophy: Aminoglycosides and Ataluren (PTC124)
Richard S. Finkel
JOURNAL OF CHILD NEUROLOGY (2010)
Genome editing with engineered zinc finger nucleases
Fyodor D. Urnov et al.
NATURE REVIEWS GENETICS (2010)
Marginal Level Dystrophin Expression Improves Clinical Outcome in a Strain of Dystrophin/Utrophin Double Knockout Mice
Dejia Li et al.
PLOS ONE (2010)
PTC124 targets genetic disorders caused by nonsense mutations
Ellen M. Welch et al.
NATURE (2007)