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Title:

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

Document type:
Review; Journal Article; Review
Author(s):
Kupatt, Christian; Windisch, Alina; Moretti, Alessandra; Wolf, Eckhard; Wurst, Wolfgang; Walter, Maggie C
Abstract:
Mutations in Dystrophin, one of the largest proteins in the mammalian body, are causative for a severe form of muscle disease, Duchenne Muscular Dystrophy (DMD), affecting not only skeletal muscle, but also the heart. In particular, exons 45-52 constitute a hotspot for DMD mutations. A variety of molecular therapies have been developed, comprising vectors encoding micro- and minidystrophins as well as utrophin, a protein with partially overlapping functions. With the advent of the CRISPR-Cas9-nu...     »
Journal title abbreviation:
Gene Ther
Year:
2021
Journal volume:
28
Journal issue:
9
Pages contribution:
542-548
Fulltext / DOI:
doi:10.1038/s41434-021-00222-4
Pubmed ID:
http://view.ncbi.nlm.nih.gov/pubmed/33531685
Print-ISSN:
0969-7128
TUM Institution:
617; Arbeitsgruppe Tissue Engineering und Regenerative Medizin; Klinik und Poliklinik für Innere Medizin I, Kardiologie
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