Hematopoietic stem cell gene therapy for the treatment of metachromatic leukodystrophy [electronic resource] / Alessandra Biffi.
Material type:
FilmSeries: Henry Stewart talksBiomedical & life sciences collection. Gene transfer and gene therapy: Publisher: London : Henry Stewart Talks, 2014Description: 1 online resource (1 streaming video file (37 min.) : color, sound)Subject(s): Brain -- Diseases -- Genetic aspects | Gene therapy | Medical genetics | Stem cells -- Therapeutic use | Genetic Therapy | Genetics, Medical | Hematopoietic Stem Cell Transplantation | Hematopoietic Stem Cells -- metabolism | Lentivirus | Leukodystrophy, Metachromatic -- genetics | Leukodystrophy, Metachromatic -- therapy | Microglia | Virus Integration -- geneticsOnline resources: Click here to access online | Series Animated audio-visual presentation with synchronized narration.
Title from title frames.
Contents: Metachromatic Leukodystrophy (MLD) -- Hematopoietic stem cell therapy (HCT) for lysosomal storage disorders (LSDs) -- Improving HCT efficacy -- The role of brain pre-conditioning -- Mechanisms of microglia turnover -- Gene therapy (GT) & enzyme release -- Gene therapy in murine models -- HSC gene therapy for MLD -- Lentivirus integration site analysis -- Stem cells analysis -- ARSA activity and delivery to the CNS -- Cerebral and peripheral demyelination -- Motor function testing (GMFM).
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Mode of access: World Wide Web.