Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy-Reference-Cited by-同舟云学术

Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy

Published:2017-04 Issue:4 Volume:28 Page:328-337
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Ertl Hildegund C.J.¹,High Katherine A.²

Affiliation:

1. Wistar Institute, Philadelphia, Pennsylvania.

2. Spark Therapeutics, Philadelphia, Pennsylvania.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2016.172

Reference99 articles.

1. Gene therapy for the hemophilias

2. Therapeutic Levels of Factor IX Expression Using a Muscle-Specific Promoter and Adeno-Associated Virus Serotype 1 Vector

3. Recombinant adeno-associated virus for muscle directed gene therapy

4. Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy

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