Hemophilia gene therapy is effective and safe
Author:
Affiliation:
1. University of Sheffield
Publisher
American Society of Hematology
Subject
Cell Biology,Hematology,Immunology,Biochemistry
Link
http://ashpublications.org/blood/article-pdf/131/9/952/1406871/blood824144.pdf
Reference6 articles.
1. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B;Nathwani;N Engl J Med,2011
2. Long-term safety and efficacy of factor IX gene therapy in hemophilia B;Nathwani;N Engl J Med,2014
3. Hemophilia B gene therapy with a high-specific activity factor IX variant;George;N Engl J Med,2017
4. AAV5-factor VIII gene transfer in severe hemophilia A;Rangarajan;N Engl J Med,2017
Cited by 4 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. Progress, and prospects in the therapeutic armamentarium of persons with congenital hemophilia. Defining the place for liver-directed gene therapy;Blood Reviews;2023-03
2. Platelet gene therapy induces robust immune tolerance even in a primed model via peripheral clonal deletion of antigen-specific T cells;Molecular Therapy - Nucleic Acids;2021-03
3. In vivo enrichment of genetically manipulated platelets for murine hemophilia B gene therapy;Journal of Cellular Physiology;2020-06-08
4. Unmasking Hemophilia B After Hip Aspiration;JBJS Case Connector;2019-04
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