Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A-Reference-Cited by-同舟云学术

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Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A

Published:2018-06 Issue: Volume:9 Page:57-69
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Brown Harrison C.,Zakas Philip M.,George Stephan N.,Parker Ernest T.,Spencer H. Trent,Doering Christopher B.

Funder

NIH/National Heart, Lung and Blood Institute

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference49 articles.

1. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector;Kay;Nat. Genet.,2000

2. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response;Manno;Nat. Med.,2006

3. 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;Nathwani;Blood,2006

4. Long-term safety and efficacy of factor IX gene therapy in hemophilia B;Nathwani;N. Engl. J. Med.,2014

5. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B;Nathwani;N. Engl. J. Med.,2011

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1. Adeno-associated virus as a delivery vector for gene therapy of human diseases;Signal Transduction and Targeted Therapy;2024-04-03

2. Shifting Paradigms and Arising Concerns in Severe Hemophilia A Treatment;Seminars in Thrombosis and Hemostasis;2024-01-15

3. Humanization and functional characterization of enhanced coagulation factor IX variants identified through ancestral sequence reconstruction;Journal of Thrombosis and Haemostasis;2023-11

4. Transplanting FVIII/ET3-secreting cells in fetal sheep increases FVIII levels long-term without inducing immunity or toxicity;Nature Communications;2023-07-14

5. Enhancing the effectiveness of γδ T cells by mRNA transfection of chimeric antigen receptors or bispecific T cell engagers;Molecular Therapy - Oncolytics;2023-06

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