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Nonviral Transfer of the Gene Encoding Coagulation Factor VIII in Patients with Severe Hemophilia A

✍ Scribed by Roth, David A.; Tawa, Nicholas E.; O'Brien, Joanne M.; Treco, Douglas A.; Selden, Richard F

Book ID
121363220
Publisher
Massachusetts Medical Society
Year
2001
Tongue
English
Weight
204 KB
Volume
344
Category
Article
ISSN
0096-6762

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Hemophilia A is a common X-linked bleeding disorder caused by various types of mutations in the factor VIII gene F8C. The most common intron 22-inversion is responsible for about 40% of the severe hemophilia A cases while large deletions, point mutations and small (less than 100 bp) deletions or ins

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Hemophilia A is a common X-linked bleeding disorder caused by mutations in the coagulation factor VIII gene. The entire coding and essential sequences of the factor VIII gene were generated by a combination of genomic DNA amplification and long reverse transcription-polymerase chain reaction (long R