Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial-Reference-Cited by-同舟云学术

Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial

Published:2015-02 Issue:2 Volume:26 Page:69-81
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Monahan Paul E.¹²³,Sun Junjiang¹,Gui Tong¹,Hu Genlin¹,Hannah William B.¹,Wichlan David G.¹²,Wu Zhijian¹,Grieger Joshua C.¹,Li Chengwen¹,Suwanmanee Thipparat¹,Stafford Darrel W.⁴,Booth Carmen J.⁵,Samulski Jade J.⁶,Kafri Tal¹⁷,McPhee Scott W.J.⁶,Samulski R. Jude¹⁸

Affiliation:

1. Gene Therapy Center, University of North Carolina, Chapel Hill, NC 27599.

2. Department of Pediatrics, University of North Carolina, Chapel Hill, NC 27599.

3. Harold R. Roberts Comprehensive Hemophilia Diagnostic and Treatment Center, University of North Carolina, Chapel Hill, NC 27599.

4. Department of Biology, University of North Carolina, Chapel Hill, NC 27599.

5. Section of Comparative Medicine, Yale University School of Medicine, New Haven, CT 06519.

6. Asklepios BioPharmaceutical, Chapel Hill, NC 27517.

7. Department of Microbiology and Immunology, University of North Carolina, Chapel Hill, NC 27599.

8. Department of Pharmacology, University of North Carolina, Chapel Hill, NC 27599.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2014.106

Reference37 articles.

1. Gene Therapy for Hemophilia: Addressing the Coming Challenges of Affordability and Accessibility

2. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

3. Immune responses to AAV vectors: overcoming barriers to successful gene therapy

4. Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

5. Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

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