Adeno-Associated Virus Gene Therapy for Liver Disease
Author:
Affiliation:
1. Dimension Therapeutics, Cambridge, Massachusetts.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
http://www.liebertpub.com/doi/pdf/10.1089/hum.2016.160
Reference125 articles.
1. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy
2. Increased apoptosis of Huntington disease lymphoblasts associated with repeat length-dependent mitochondrial depolarization
3. Sustained Expression of Therapeutic Level of Factor IX in Hemophilia B Dogs by AAV-Mediated Gene Therapy in Liver
4. Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs
5. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
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