Evidence of Multiyear Factor IX Expression by AAV-Mediated Gene Transfer to Skeletal Muscle in an Individual with Severe Hemophilia B-Reference-Cited by-同舟云学术

Evidence of Multiyear Factor IX Expression by AAV-Mediated Gene Transfer to Skeletal Muscle in an Individual with Severe Hemophilia B

Published:2006-09 Issue:3 Volume:14 Page:452-455
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Jiang Haiyan,Pierce Glenn F.,Ozelo Margareth C.,de Paula Erich V.,Vargas Joseph A.,Smith Peter,Sommer Jürg,Luk Alvin,Manno Catherine S.,High Katherine A.,Arruda Valder R.

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference17 articles.

1. Prophylactic infusion regimens in the management of hemophilia;Ljung;Thromb. Haemostasis,1999

2. Haemophilia prophylaxis in young patients—A long term follow up;Lofqvist;J. Intern. Med.,1997

3. Posttranslational modifications of recombinant myotube-synthesized human Factor IX;Arruda;Blood,2001

4. Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia;Monahan;Gene Ther.,1998

5. Long-term correction of canine hemophilia B by gene transfer of blood coagulation Factor IX mediated by adeno-associated viral vector;Herzog;Nat. Med.,1999

Cited by 186 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. The evolving landscape of gene therapy for congenital severe hemophilia: a 2024 state of the art;Bleeding, Thrombosis and Vascular Biology;2024-09-04

2. Hemophilia Healing with AAV: Navigating the Frontier of Gene Therapy;Current Gene Therapy;2024-08

3. Recent Advances in Gene Therapy for Hemophilia: Projecting the Perspectives;Biomolecules;2024-07-15

4. AAV mediated gene therapy for haemophilia B: From the early attempts to modern trials;Thrombosis Research;2024-04

5. Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia;Journal of Thrombosis and Haemostasis;2023-12