Gene therapy for Hemophilia [electronic resource] / Katherine High.
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 (56 min.) : color, sound)Subject(s): Gene therapy | Genetic transformation | Genetic vectors -- Therapeutic use | Hemophilia -- Gene therapy | Viral genetics | Dependovirus -- genetics | Gene Transfer Techniques | Genetic Therapy -- methods | Genetic Vectors -- therapeutic use | Hemophilia A -- genetics | Hemophilia A -- therapy | Hemophilia B -- genetics | Hemophilia B -- therapyOnline resources: Click here to access online | SeriesAnimated audio-visual presentation with synchronized narration.
Title from title frames.
Contents: Hemophilia treatment past and present -- Gene transfer for genetic disease -- Viruses engineered to deliver genes -- Gene therapy using integrating vectors -- Adeno-associated virus (AAV) structure and composition -- AAV-Factor IX vectors -- AAV-mediated gene transfer for hemophilia -- Risk of germline transmission -- Peptide library based on AAV and Factor IX -- Transaminitis and liver enzymes -- Neutralizing antibodies to AAV -- Self-complementary DNA and Factor IX synthesis -- Strategies to overcome immunity to AAV -- Use of empty capsids.
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Mode of access: World Wide Web.