Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
About this item
Full title
Author / Creator
George, Lindsey A , Monahan, Paul E , Eyster, M. Elaine , Sullivan, Spencer K , Ragni, Margaret V , Croteau, Stacy E , Rasko, John E.J , Recht, Michael , Samelson-Jones, Benjamin J , MacDougall, Amy , Jaworski, Kristen , Noble, Robert , Curran, Marla , Kuranda, Klaudia , Mingozzi, Federico , Chang, Tiffany , Reape, Kathleen Z , Anguela, Xavier M and High, Katherine A
Publisher
United States: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
United States: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
At a median follow-up of 3.5 years after gene therapy with a factor VIII–containing adeno-associated virus, 16 of 18 men with hemophilia A had enough factor VIII expression that bleeding events were reduced to nearly zero and prophylaxis was discontinued. Vector-related hepatitis led to elimination of the vector in 2 men, but immunosuppressive ther...
Alternative Titles
Full title
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
Authors, Artists and Contributors
Author / Creator
Monahan, Paul E
Eyster, M. Elaine
Sullivan, Spencer K
Ragni, Margaret V
Croteau, Stacy E
Rasko, John E.J
Recht, Michael
Samelson-Jones, Benjamin J
MacDougall, Amy
Jaworski, Kristen
Noble, Robert
Curran, Marla
Kuranda, Klaudia
Mingozzi, Federico
Chang, Tiffany
Reape, Kathleen Z
Anguela, Xavier M
High, Katherine A
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8672712
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8672712
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa2104205
