High, Katherine A., 1951-

Gene therapy for Hemophilia [electronic resource] / Katherine High. - London : Henry Stewart Talks, 2014. - 1 online resource (1 streaming video file (56 min.) : color, sound). - Gene transfer and gene therapy, 2056-452X . - Henry Stewart talks. Biomedical & life sciences collection. Gene transfer and gene therapy. .

Animated 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.

3745 Henry Stewart Talks


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--therapy.
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