Genome editing for Duchenne muscular dystrophy: a glimpse of the future?

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy

(2020) A. Moretti et al.   NATURE MEDICINE

Effect of Ataluren on dystrophin mutations

(2020) Joachim Berger et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy

(2020) Kathryn R. Wagner et al.   NEUROMUSCULAR DISORDERS

Progressive Proteome Changes in the Myocardium of a Pig Model for Duchenne Muscular Dystrophy

(2020) Hathaichanok Tamiyakul et al.   iScience

Favorable outcomes of COVID-19 in recipients of hematopoietic cell transplantation

(2020) Gunjan L. Shah et al.   JOURNAL OF CLINICAL INVESTIGATION

rAAVrh74.MCK.GALGT2 Protects Against Loss of Hemodynamic Function in the Aging mdx Mouse Heart

(2019) Rui Xu et al.   MOLECULAR THERAPY

Therapeutic developments for Duchenne muscular dystrophy

(2019) Ingrid E. C. Verhaart et al.   Nature Reviews Neurology

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

(2019) Eric P. Hoffman et al.   NEUROLOGY

Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy

(2019) Laurent Servais et al.   NEUROMUSCULAR DISORDERS

Detection of collagens by multispectral optoacoustic tomography as an imaging biomarker for Duchenne muscular dystrophy

(2019) Adrian P. Regensburger et al.   NATURE MEDICINE

Exon-skipping advances for Duchenne muscular dystrophy

(2018) Lucía Echevarría et al.   HUMAN MOLECULAR GENETICS

Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan

(2018) David J Birnkrant et al.   LANCET NEUROLOGY

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged ≥2 to

(2018) R. Kong et al.   NEUROMUSCULAR DISORDERS

Modeling lethal X-linked genetic disorders in pigs with ensured fertility

(2018) Hitomi Matsunari et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

(2018) Leonela Amoasii et al.   SCIENCE

Emerging Strategies in the Treatment of Duchenne Muscular Dystrophy

(2018) Perry B. Shieh   Neurotherapeutics

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

(2017) Craig M McDonald et al.   LANCET

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

(2017) Caroline Le Guiner et al.   Nature Communications

Histological effects of givinostat in boys with Duchenne muscular dystrophy

(2016) Paolo Bettica et al.   NEUROMUSCULAR DISORDERS

Progressive muscle proteome changes in a clinically relevant pig model of Duchenne muscular dystrophy

(2016) Thomas Fröhlich et al.   Scientific Reports

Crystal Structure of Staphylococcus aureus Cas9

(2015) Hiroshi Nishimasu et al.   CELL

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Development of an intein-mediated split–Cas9 system for gene therapy

(2015) Dong-Jiunn Jeffery Truong et al.   NUCLEIC ACIDS RESEARCH

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

(2014) Randall J. Platt et al.   CELL

Ataluren treatment of patients with nonsense mutation dystrophinopathy

(2014) Katharine Bushby et al.   MUSCLE & NERVE

The burden of Duchenne muscular dystrophy: An international, cross-sectional study

(2014) E. Landfeldt et al.   NEUROLOGY

Comparative cost of illness analysis and assessment of health care burden of Duchenne and Becker muscular dystrophies in Germany

(2014) Olivia Schreiber-Katz et al.   Orphanet Journal of Rare Diseases

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

(2014) C. Long et al.   SCIENCE

Structures of Cas9 Endonucleases Reveal RNA-Mediated Conformational Activation

(2014) M. Jinek et al.   SCIENCE

Adeno-associated virus: fit to serve

(2014) Eric Zinn et al.   Current Opinion in Virology

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Reducing CTGF/CCN2 slows down mdx muscle dystrophy and improves cell therapy

(2013) Maria Gabriela Morales et al.   HUMAN MOLECULAR GENETICS

Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle

(2013) Nikolai Klymiuk et al.   HUMAN MOLECULAR GENETICS

Faster Protein Splicing with theNostoc punctiformeDnaE Intein Using Non-native Extein Residues

(2013) Manoj Cheriyan et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy

(2013) Jin-Hong Shin et al.   MOLECULAR THERAPY

Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

(2013) Richard S. Finkel et al.   PLoS One

Long-Term Preservation of Cardiac Structure and Function After Adeno-Associated Virus Serotype 9-Mediated Microdystrophin Gene Transfer in mdx Mice

(2012) Stefanie Schinkel et al.   HUMAN GENE THERAPY

Kinetic Control of One-Pot Trans-Splicing Reactions by Using a Wild-Type and Designed Split Intein

(2011) Neel H. Shah et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Protein Trans-Splicing as a Means for Viral Vector-Mediated In Vivo Gene Therapy

(2008) Juan Li et al.   HUMAN GENE THERAPY