Dystrophin Gene-Editing Stability Is Dependent on Dystrophin Levels in Skeletal but Not Cardiac Muscles

Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy

(2020) Kasia Dzierlega et al.   GENE THERAPY

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

(2019) Rui Xu et al.   MOLECULAR THERAPY

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

Development of Novel Micro-dystrophins with Enhanced Functionality

(2019) Julian N. Ramos et al.   MOLECULAR THERAPY

In Situ Modification of Tissue Stem and Progenitor Cell Genomes

(2019) Jill M. Goldstein et al.   Cell Reports

AAV-based gene therapies for the muscular dystrophies

(2019) Julie M Crudele et al.   HUMAN MOLECULAR GENETICS

High levels of AAV vector integration into CRISPR-induced DNA breaks

(2019) Killian S. Hanlon et al.   Nature Communications

CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo

(2018) Benjamin L. Duchêne et al.   MOLECULAR THERAPY

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

(2018) Leonela Amoasii et al.   SCIENCE

Cas9 immunity creates challenges for CRISPR gene editing therapies

(2018) Julie M. Crudele et al.   Nature Communications

Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/mdx Mice

(2018) Tahnee L. Kennedy et al.   Molecular Therapy-Methods & Clinical Development

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

(2017) Leonela Amoasii et al.   Science Translational Medicine

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

(2017) Caroline Le Guiner et al.   Nature Communications

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

(2017) Niclas E. Bengtsson et al.   Nature Communications

Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles

(2016) Cécile Peccate et al.   HUMAN MOLECULAR GENETICS

Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method

(2016) Jean-Paul Iyombe-Engembe et al.   Molecular Therapy-Nucleic Acids

Progress and prospects of gene therapy clinical trials for the muscular dystrophies

(2015) Niclas E. Bengtsson et al.   HUMAN MOLECULAR GENETICS

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

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

Muscular dystrophy in the mdx mouse is a severe myopathy compounded by hypotrophy, hypertrophy and hyperplasia

(2015) William Duddy et al.   Skeletal Muscle

Adeno-associated viral vectors do not efficiently target muscle satellite cells

(2014) Andrea LH Arnett et al.   Molecular Therapy-Methods & Clinical Development

Evidence-based path to newborn screening for duchenne muscular dystrophy

(2012) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

The Polyproline Site in Hinge 2 Influences the Functional Capacity of Truncated Dystrophins

(2010) Glen B. Banks et al.   PLoS Genetics

DIAPHRAGM MUSCLE STRIP PREPARATION FOR EVALUATION OF GENE THERAPIES IN mdx MICE

(2008) John A Faulkner et al.   CLINICAL AND EXPERIMENTAL PHARMACOLOGY AND PHYSIOLOGY

Microutrophin Delivery Through rAAV6 Increases Lifespan and Improves Muscle Function in Dystrophic Dystrophin/Utrophin-deficient Mice

(2008) Guy L Odom et al.   MOLECULAR THERAPY

Systemic Microdystrophin Gene Delivery Improves Skeletal Muscle Structure and Function in Old Dystrophic mdx Mice

(2008) Paul Gregorevic et al.   MOLECULAR THERAPY