Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice-Reference-Cited by-同舟云学术

Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice

Published:2017-05 Issue:5 Volume:28 Page:392-402
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Greig Jenny A.¹,Wang Qiang¹,Reicherter Amanda L.¹,Chen Shu-Jen¹,Hanlon Alexandra L.²,Tipper Christopher H.³,Clark K. Reed³,Wadsworth Samuel³,Wang Lili⁴,Wilson James M.¹

Affiliation:

1. Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.

2. School of Nursing, University of Pennsylvania, Philadelphia, Pennsylvania.

3. Dimension Therapeutics, Cambridge, Massachusetts.

4. Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2016.128

Reference30 articles.

1. Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

2. Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

3. Codon optimization of human factor VIII cDNAs leads to high-level expression

4. Novel Forms of B-Domain-Deleted Recombinant Factor VIII Molecules. Construction and Biochemical Characterization

5. Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

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