Impending therapies for Duchenne muscular dystrophy
TA Partridge - Current opinion in neurology, 2011 - journals.lww.com
Although genetic and cell-mediated approaches are currently showing genuine promise in
preclinical and clinical trials, there remains considerable interest in the development of …
preclinical and clinical trials, there remains considerable interest in the development of …
Duchenne muscular dystrophy and dystrophin: Pathogenesis and opportunities for treatment: Third in molecular medicine review series
Duchenne muscular dystrophy (DMD) is caused by mutations in the gene that encodes the
427‐kDa cytoskeletal protein dystrophin. Increased knowledge of the function of dystrophin …
427‐kDa cytoskeletal protein dystrophin. Increased knowledge of the function of dystrophin …
Progress toward gene therapy for Duchenne muscular dystrophy
JR Chamberlain, JS Chamberlain - Molecular Therapy, 2017 - cell.com
Duchenne muscular dystrophy (DMD) has been a major target for gene therapy
development for nearly 30 years. DMD is among the most common genetic diseases, and …
development for nearly 30 years. DMD is among the most common genetic diseases, and …
Technology insight: therapy for Duchenne muscular dystrophy—an opportunity for personalized medicine?
LE Lim, TA Rando - Nature Clinical Practice Neurology, 2008 - nature.com
Since the identification of dystrophin as the protein product of the Duchenne and Becker
muscular dystrophy locus, many different mutations, encompassing the entire spectrum of …
muscular dystrophy locus, many different mutations, encompassing the entire spectrum of …
Evolving therapeutic options for the treatment of duchenne muscular dystrophy
ES D'Ambrosio, JR Mendell - Neurotherapeutics, 2023 - Elsevier
Duchenne muscular dystrophy (DMD) is the most common childhood form of muscular
dystrophy. It is caused by mutations in the DMD gene, leading to reduced or absent …
dystrophy. It is caused by mutations in the DMD gene, leading to reduced or absent …
Therapeutic approaches for Duchenne muscular dystrophy
TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …
Personalized gene and cell therapy for Duchenne muscular dystrophy
F Barthélémy, N Wein - Neuromuscular Disorders, 2018 - Elsevier
Dystrophinopathies are diseases caused by mutations in the Duchenne Muscular Dystrophy
gene (DMD) encoding the dystrophin protein. Depending on the type of mutation, patients …
gene (DMD) encoding the dystrophin protein. Depending on the type of mutation, patients …
Update on the treatment of Duchenne muscular dystrophy
LR Rodino-Klapac, JR Mendell, Z Sahenk - Current neurology and …, 2013 - Springer
Duchenne muscular dystrophy is the most severe childhood form of muscular dystrophy
caused by mutations in the gene responsible for dystrophin production. There is no cure …
caused by mutations in the gene responsible for dystrophin production. There is no cure …
Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches
RJ Fairclough, MJ Wood, KE Davies - Nature Reviews Genetics, 2013 - nature.com
Duchenne muscular dystrophy (DMD) is a devastating progressive disease for which there
is currently no effective treatment except palliative therapy. There are several promising …
is currently no effective treatment except palliative therapy. There are several promising …
Molecular therapeutic strategies targeting Duchenne muscular dystrophy
JR Mendell, LR Rodino-Klapac… - Journal of child …, 2010 - journals.sagepub.com
Since the discovery of the gene for Duchenne muscular dystrophy more than 20 years ago,
scientists have worked to apply molecular principles for restoration of the dystrophin protein …
scientists have worked to apply molecular principles for restoration of the dystrophin protein …