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The development and evolution of human gene therapy

Author: Theodore Friedmann
Publisher: London : Henry Stewart Talks, 2010.
Series: Henry Stewart talks., Biomedical & life sciences collection., Introduction to human genetics : fundamentals and latest advances.
Edition/Format:   eVideo : Clipart/images/graphics : EnglishView all editions and formats
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Material Type: Clipart/images/graphics, Internet resource, Videorecording
Document Type: Internet Resource, Computer File, Visual material
All Authors / Contributors: Theodore Friedmann
OCLC Number: 720056283
Notes: Animated audio-visual presentation with synchronized narration.
Title from title frames.
Description: 1 streaming video file (29 min.) : digital, mono, SWF file, sound, color.
Details: Mode of access: World Wide Web.; System requirements: Operating System: PC Windows 2000+, Mac OSX+ 3.2. Browser Compatibility: IE6+, Firefox 2+, Opera 9+, Safari 2+ 3.3. Browser settings: enable JavaScript, enable popups from the Henry Stewart Talks site. 3.4. Required Browser Plugins & Viewers: Adobe (Macromedia) Flash Player 7+, Adobe Acrobat Reader 6.0+.
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.
Series Title: Henry Stewart talks., Biomedical & life sciences collection., Introduction to human genetics : fundamentals and latest advances.
Responsibility: Theodore Friedmann.
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