Exon Snipping in Duchenne Muscular Dystrophy

Exon Snipping in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a life-limiting neuromuscular disorder caused by mutations in the DMD gene encoding dystrophin. We discuss very recent studies that used CRISPR/Cas9 technology to ‘snip out’ mutated exons in DMD , restoring the reading frame of the gene. We also present cautionar...

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Veröffentlicht in:Trends in molecular medicine 2016-03, Vol.22 (3), p.187-189
Hauptverfasser: Kemaladewi, Dwi U, Cohn, Ronald D
Format: Artikel
Sprache:eng
Schlagworte:
Animals
CRISPR-Cas Systems
Dystrophin - genetics
Exons - genetics
Genetic Therapy - methods
Male
Muscle, Skeletal - metabolism
Muscular Dystrophy, Duchenne - therapy
Pathology
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Beschreibung
Zusammenfassung:Duchenne muscular dystrophy (DMD) is a life-limiting neuromuscular disorder caused by mutations in the DMD gene encoding dystrophin. We discuss very recent studies that used CRISPR/Cas9 technology to ‘snip out’ mutated exons in DMD , restoring the reading frame of the gene. We also present cautionary aspects of translating this exciting technology into clinical practice.
ISSN:1471-4914
1471-499X
DOI:10.1016/j.molmed.2016.01.007