A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells-Reference-Cited by-同舟云学术

A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells

Published:2020-11-16 Issue:1 Volume:39 Page:47-55
ISSN:1087-0156
Container-title:Nature Biotechnology
language:en
Short-container-title:Nat Biotechnol

Author:

Nguyen Giang N.,Everett John K.,Kafle Samita,Roche Aoife M.,Raymond Hayley E.,Leiby Jacob,Wood Christian,Assenmacher Charles-Antoine^ORCID,Merricks Elizabeth P.,Long C. Tyler,Kazazian Haig H.,Nichols Timothy C.,Bushman Frederic D.^ORCID,Sabatino Denise E.^ORCID

Publisher

Springer Science and Business Media LLC

Subject

Biomedical Engineering,Molecular Medicine,Applied Microbiology and Biotechnology,Bioengineering,Biotechnology

Link

http://www.nature.com/articles/s41587-020-0741-7.pdf

Reference56 articles.

1. Rangarajan, S. et al. AAV5-factor VIII gene transfer in severe hemophilia A. N. Engl. J. Med. 377, 2519–2530 (2017).

2. High, K. A. et al. A phase 1/2 trial of investigational Spk-8011 in hemophilia A demonstrates durable expression and prevention of bleeds. Blood 132, 487 (2018).

3. Nathwani, A. C. et al. GO-8: preliminary results of a phase I/II dose escalation trial of gene therapy for haemophilia A using a novel human factor VIII variant. Blood 132, 489 (2018).

4. Pasi, K. J. et al. Multiyear follow-up of AAV5-hFVIII-SQ gene therapy for hemophilia A. N. Engl. J. Med. 382, 29–40 (2020).

5. Jiang, H. et al. Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs. Blood 108, 107–115 (2006).

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