Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

Nathwani, Amit C

Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. [electronic resource] - Molecular therapy : the journal of the American Society of Gene Therapy May 2011 - 876-85 p. digital

Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

1525-0024

10.1038/mt.2010.274 doi


Animals
Capsid Proteins--genetics
Dependovirus--genetics
Factor IX--genetics
Gene Expression
Genetic Therapy--methods
Genetic Vectors
HEK293 Cells
Hemophilia B--genetics
Humans
In Situ Hybridization, Fluorescence
Liver--metabolism
Macaca
Mice