AAV5–Factor VIII Gene Transfer in Severe Hemophilia A
AAV5–Factor VIII Gene Transfer in Severe Hemophilia A
About this item
Full title
AAV5–Factor VIII Gene Transfer in Severe Hemophilia A
Author / Creator
Publisher
United States: Massachusetts Medical Society
Journal title
The New England journal of medicine, 2017-12, Vol.377 (26), p.2519-2530
Language
English
Formats
Publication information
Publisher
United States: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
An AAV5 vector with a functional factor VIII gene was tested in men with hemophilia A. Men receiving the high dose had a factor VIII level above 5 IU per deciliter at 1 year; most had a normal level. Toxic effects were minimal, and bleeding episodes nearly eliminated.
Alternative Titles
Full title
AAV5–Factor VIII Gene Transfer in Severe Hemophilia A
Authors, Artists and Contributors
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_1975595688
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_1975595688
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa1708483
