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2006, Neurological disease and therapy, ISBN 0824723252, Volume 79., xxii, 461
Duchenne Muscular Dystrophy (DMD) is one of the most prevalent genetic disorders of childhood and currently stands as an incurable condition. This... 
Treatment | Muscular Dystrophy, Duchenne | Duchenne muscular dystrophy | therapy | Medical Genetics | Neurology | Neuroscience | Muscular Dystrophy, Duchenne - therapy
Book
2007, Protein epidemiology : understanding human diseases at the level of protein structure and function
Web Resource
2007, Protein epidemiology : understanding human diseases at the level of protein structure and function
Web Resource
Human Molecular Genetics, ISSN 0964-6906, 06/2019
Journal Article
2008, ISBN 0123744547, Volume 84
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy. There is no effective treatment and patients typically die in approximately... 
Book Chapter
Methods in Molecular Biology, ISSN 1064-3745, 2018, Volume 1687, pp. 257 - 266
Journal Article
2006, Neurological disease and therapy, ISBN 9780824723255, Volume 79
Web Resource
Science Translational Medicine, ISSN 1946-6234, 11/2010, Volume 2, Issue 57
Journal Article
Nature Communications, ISSN 2041-1723, 02/2017, Volume 8, Issue 1, pp. 14454 - 14454
Gene replacement therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne muscular dystrophy (DMD). A related approach... 
CRISPR-CAS9 | SKELETAL | EXPRESSION | VECTORS | MULTIDISCIPLINARY SCIENCES | MDX MICE | Index Medicus
Journal Article
Cell, ISSN 0092-8674, 12/2010, Volume 143, Issue 7, pp. 1040 - 1042
The lack of appropriate animal models has hampered efforts to develop therapies for Duchenne muscular dystrophy (DMD). A new mouse model lacking both... 
CELLS | DEFICIENT MICE | BIOCHEMISTRY & MOLECULAR BIOLOGY | CELL BIOLOGY | Telomerase | Dystrophin | Analysis | Utrophin | Duchenne muscular dystrophy
Journal Article
Molecular Therapy, ISSN 1525-0016, 10/2013, Volume 21, Issue 10, pp. 1821 - 1822
Journal Article
Muscle & Nerve, ISSN 0148-639X, 05/2013, Volume 47, Issue 5, pp. 649 - 663
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2015, Volume 23, p. S245
Journal Article
Science, ISSN 0036-8075, 12/2012, Volume 338, Issue 6113, pp. 1431 - 1432
  Establishing dystrophin as the mutated gene in Duchenne muscular dystrophy (DMD) was arguably the first successful use of genetic information to identify a... 
Messenger RNA | Splicing | Exons | Genes | Duchenne muscular dystrophy | Medical genetics | Stem cells | Cell lines | PERSPECTIVES | Antisense oligonucleotides | Human genetics | DUCHENNE MUSCULAR-DYSTROPHY | MUTATIONS | MOLECULAR-BASIS | EXPRESSION | MULTIDISCIPLINARY SCIENCES | DELETION | Clinical trials | Genetic engineering | Genetic disorders | Muscular dystrophy
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