The delivery challenge: fulfilling the promise of therapeutic genome editing-Reference-Cited by-同舟云学术

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The delivery challenge: fulfilling the promise of therapeutic genome editing

Published:2020-06-29 Issue:7 Volume:38 Page:845-855
ISSN:1087-0156
Container-title:Nature Biotechnology
language:en
Short-container-title:Nat Biotechnol

Author:

van Haasteren Joost^ORCID,Li Jie,Scheideler Olivia J.,Murthy Niren,Schaffer David V.^ORCID

Funder

National Science Foundation

U.S. Department of Health & Human Services | National Institutes of Health

Innovative Genomics Institute

Gilbert Family Foundation, Chan Zuckerberg Biohub, Innovative Genomics Institute

Publisher

Springer Science and Business Media LLC

Subject

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

Link

http://www.nature.com/articles/s41587-020-0565-5.pdf

Reference139 articles.

1. Boycott, K. M., Vanstone, M. R., Bulman, D. E. & MacKenzie, A. E. Rare-disease genetics in the era of next-generation sequencing: discovery to translation. Nat. Rev. Genet. 14, 681–691 (2013).

2. Nathwani, A. C. et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N. Engl. J. Med. 371, 1994–2004 (2014).

3. Hoggatt, J. Gene therapy for “bubble boy” disease. Cell 166, 263 (2016).

4. Maguire, A. M. et al. Safety and efficacy of gene transfer for Leber’s congenital amaurosis. N. Engl. J. Med. 358, 2240–2248 (2008).

5. Choo, K. H., Gould, K. G., Rees, D. J. & Brownlee, G. G. Molecular cloning of the gene for human anti-haemophilic factor IX. Nature 299, 178–180 (1982).

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