Our Journey to Successful Gene Therapy for Hemophilia B-Reference-Cited by-同舟云学术

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Our Journey to Successful Gene Therapy for Hemophilia B

Published:2014-11 Issue:11 Volume:25 Page:923-926
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Nathwani Amit C.¹²³,Nienhuis Arthur W.⁴,Davidoff Andrew M.⁵

Affiliation:

1. Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free NHS Foundation Trust, London NW3 2QG, United Kingdom.

2. Department of Haematology, UCL Cancer Institute, London WC1E 6DD, United Kingdom.

3. National Health Services Blood and Transplant, Hertfordshire WD24 4QN, United Kingdom.

4. Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105.

5. Department of Surgery, St. Jude Children's Research Hospital, Memphis, TN 38105.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2014.2540

Reference33 articles.

1. Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial

2. Mortality rates, life expectancy, and causes of death in people with hemophilia A or B in the United Kingdom who were not infected with HIV

3. Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

4. Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock

Cited by 14 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. Challenges in development and authorisation of gene therapy products;Biological Products. Prevention, Diagnosis, Treatment;2022-03-10

3. Gene therapy and editing in the treatment of hereditary blood disorders: Medical and ethical aspects;Clinical Ethics;2021-11-06

4. Current Clinical Applications of In Vivo Gene Therapy with AAVs;Molecular Therapy;2021-02

5. Validation of an IFN-gamma ELISpot assay to measure cellular immune responses against viral antigens in non-human primates;Gene Therapy;2021-01-11

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