Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system-Reference-Cited by-同舟云学术

Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system

Published:2018-04-06 Issue:1 Volume:9 Page:
ISSN:1757-6512
Container-title:Stem Cell Research & Therapy
language:en
Short-container-title:Stem Cell Res Ther

Author:

Lyu Cuicui,Shen Jun,Wang Rui,Gu Haihui,Zhang Jianping,Xue Feng,Liu Xiaofan,Liu Wei,Fu Rongfeng,Zhang Liyan,Li Huiyuan,Zhang Xiaobing,Cheng Tao,Yang Renchi,Zhang Lei

Funder

National Natural Science Foundation of China

Tianjin Municipal Science and Technology Commission Grant

CAMS Initiative for Innovative Medicine

Novo Nordisk Hemophilia Research Fund

the Ministry of Science and Technology of China

Publisher

Springer Science and Business Media LLC

Subject

Cell Biology,Biochemistry, Genetics and Molecular Biology (miscellaneous),Molecular Medicine,Medicine (miscellaneous)

Link

http://link.springer.com/content/pdf/10.1186/s13287-018-0839-8.pdf

Reference48 articles.

1. Nienhuis AW, Nathwani AC, Davidoff AM. Gene therapy for hemophilia. Mol Ther. 2017;25(5):1163–7.

2. 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(21):1994–2004.

3. Mali P, Yang L, Esvelt KM, Aach J, Guell M, DiCarlo JE, et al. RNA-guided human genome engineering via Cas9. Science. 2013;339(6121):823–6.

4. Cong L, Ran FA, Cox D, Lin S, Barretto R, Habib N, et al. Multiplex genome engineering using CRISPR/Cas systems. Science. 2013;339(6121):819–23.

5. Xue HY, Ji LJ, Gao AM, Liu P, He JD, Lu XJ. CRISPR-Cas9 for medical genetic screens: applications and future perspectives. J Med Genet. 2016;53(2):91–7.

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