Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy-Reference-Cited by-同舟云学术

Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy

Published:2010-11 Issue:11 Volume:18 Page:1983-1994
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Hurlbut Gregory D,Ziegler Robin J,Nietupski Jennifer B,Foley Joseph W,Woodworth Lisa A,Meyers Elizabeth,Bercury Scott D,Pande Nilesh N,Souza David W,Bree Mark P,Lukason Michael J,Marshall John,Cheng Seng H,Scheule Ronald K

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

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4. AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease;McEachern;J Gene Med,2006

5. Sustained correction of bleeding disorder in hemophilia B mice by gene therapy;Wang;Proc Natl Acad Sci USA,1999

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