The development and evolution of human gene therapy [electronic resource] / Theodore Friedmann.
Material type:
FilmSeries: Henry Stewart talksBiomedical & life sciences collection. Introduction to human genetics : fundamentals and latest advances: Publisher: London : Henry Stewart Talks, 2010Description: 1 online resource (1 streaming video file (29 min.) : color, sound)Subject(s): Gene therapy | Human genetics | Medical genetics | Gene Therapy | Genetics, MedicalOnline resources: Click here to access online | Series Animated audio-visual presentation with synchronized narration.
Title from title frames.
Contents: Background in genetic concepts -- Application by Sir Archibald Garrod to human disease -- Discovery of the structure of DNA and new opportunities to understand and manipulate genes -- Proof of principle of gene transfer into human cells using the Lesch Nyhan disease model -- Rapid expansion of clinical gene therapy trials, high level of optimism and expectation -- Major setback in human OTC gene therapy study -- Patient death -- Scientific and clinical success with another disease model -- Severe combined immunodeficiency (SCID) -- Setback in SCID -- Leukemia in some treated patients -- Additional clinical successes, especially in Leber's congenital blindness.
Access restricted to subscribers.
Mode of access: World Wide Web.