Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B-Reference-Cited by-同舟云学术

Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B

Published:2020-10-01 Issue:19-20 Volume:31 Page:1114-1123
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Brown Harrison C.¹,Doering Christopher B.²,Herzog Roland W.³,Ling Chen⁴,Markusic David M.³,Spencer H. Trent²,Srivastava Alok⁵,Srivastava Arun⁶

Affiliation:

1. Expression Therapeutics, Tucker, Georgia, USA.

2. Department of Pediatrics, Aflac Cancer and Blood Disorders Center, Emory University School of Medicine, Atlanta, Georgia, USA.

3. Department of Pediatrics, Herman B Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis, Indiana, USA.

4. State Key Laboratory of Genetic Engineering, School of Life Sciences, Fudan University, Shanghai, China.

5. Department of Haematology, Christian Medical College and Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Vellore, Tamil Nadu, India.

6. Division of Cellular and Molecular Therapy, Departments of Pediatrics and Molecular Genetics and Microbiology, Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, Florida, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2020.099

Reference35 articles.

1. Adeno-associated virus 2-mediated gene transfer in vivo: organ-tropism and expression of transduced sequences in mice

2. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

3. Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors

4. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

5. Adeno-Associated Virus-Mediated Gene Transfer of Factor IX for Treatment of Hemophilia B by Gene Therapy

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