CRISPR/Cas9-mediated gene correction in hemophilia B patient-derived iPSCs-Reference-Cited by-同舟云学术

CRISPR/Cas9-mediated gene correction in hemophilia B patient-derived iPSCs

Published:2019-10-29 Issue:2 Volume:111 Page:225-233
ISSN:0925-5710
Container-title:International Journal of Hematology
language:en
Short-container-title:Int J Hematol

Author:

Morishige Satoshi,Mizuno Shinichi,Ozawa Hidetoshi,Nakamura Takayuki,Mazahery Ahmad,Nomura Kei,Seki Ritsuko,Mouri Fumihiko,Osaki Koichi,Yamamura Kenichi,Okamura Takashi,Nagafuji Koji^ORCID

Publisher

Springer Science and Business Media LLC

Subject

Hematology

Link

http://link.springer.com/content/pdf/10.1007/s12185-019-02765-0.pdf

Reference24 articles.

1. Li T, Miller CH, Payne AB, Craig HW. The CDC Hemophilia B mutation project mutation list: a new online resource. Mol Genet Genomic Med. 2013;1:238–45.

2. George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, et al. Hemophilia B gene therapy with a high-specific-activity factor IX variant. N Engl J Med. 2017;377:2215–27.

3. Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014;371:1994–2004.

4. Patel N, Reiss U, Davidoff AM, Nathwani AC. Progress towards gene therapy for haemophilia B. Int J Hematol. 2014;99:372–6.

5. Ohmori T. Advances in gene therapy for hemophilia: basis, current status, and future perspectives. Int J Hematol. 2018; https://doi.org/10.1007/s12185-018-2513-4 .

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