Gene therapy for the muscular dystrophies [electronic resource] / Jeffrey S. Chamberlain.
Material type:
FilmSeries: Henry Stewart talksBiomedical & life sciences collection. Gene transfer and gene therapy: ; Henry Stewart talksBiomedical & life sciences collection. Molecular genetics of human disease: Publisher: London : Henry Stewart Talks, 2014Description: 1 online resource (1 streaming video file (66 min.) : color, sound)Subject(s): Duchenne muscular dystrophy -- Gene therapy | Duchenne muscular dystrophy -- Genetic aspects | Gene therapy | Genetic vectors -- Therapeutic use | Human molecular genetics | Medical genetics | Muscular dystrophy -- Gene therapy | Muscular dystrophy -- Genetic aspects | Genetic Therapy | Genetic Vectors | Muscular Dystrophies -- genetics | Muscular Dystrophies -- therapy | Muscular Dystrophy, Duchenne -- genetics | Muscular Dystrophy, Duchenne -- therapyOnline resources: Click here to access online | Series (Gene transfer and gene therapy) | Series (Molecular genetics of human disease) Animated audio-visual presentation with synchronized narration.
Title from title frames.
Contents: Introduction to the muscular dystrophies -- Duchenne muscular dystrophy (DMD) -- Dystrophin, the gene product of the DMD locus -- The molecular basis of DMD -- Strategies for the development of DMD therapies -- Adeno-associated viral (AAV) vectors and micro-dystrophins -- Scaling gene therapies to large animal models -- Exon skipping therapies.
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Mode of access: World Wide Web.