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AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects

โœ Scribed by Hasbrouck, N C; High, K A


Book ID
109855181
Publisher
Nature Publishing Group
Year
2008
Tongue
English
Weight
133 KB
Volume
15
Category
Article
ISSN
0969-7128

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Gene therapy is being explored as a means of treating a variety of genetic diseases. Because of the central role of the liver in many metabolic pathways, and the relative ease with which foreign genes can be delivered to hepatocytes, monogenic disorders that result in deficiencies of liver-specific