Genome editing of HBG1 and HBG2 to induce fetal hemoglobin

Genome editing of HBG1 and HBG2 to induce fetal hemoglobin

Abstract: Induction of fetal hemoglobin (HbF) via clustered regularly interspaced short palindromic repeats/Cas9–mediated disruption of DNA regulatory elements that repress γ-globin gene (HBG1 and HBG2) expression is a promising therapeutic strategy for sickle cell disease (SCD) and β-thalassemia, a...

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Main Authors: Jean-Yves Métais, Phillip A. Doerfler, Thiyagaraj Mayuranathan, Daniel E. Bauer, Stephanie C. Fowler, Matthew M. Hsieh, Varun Katta, Sagar Keriwala, Cicera R. Lazzarotto, Kevin Luk, Michael D. Neel, S. Scott Perry, Samuel T. Peters, Shaina N. Porter, Byoung Y. Ryu, Akshay Sharma, Devlin Shea, John F. Tisdale, Naoya Uchida, Scot A. Wolfe, Kaitly J. Woodard, Yuxuan Wu, Yu Yao, Jing Zeng, Shondra Pruett-Miller, Shengdar Q. Tsai, Mitchell J. Weiss
格式: Article
語言:English
出版: Elsevier 2019-11-01
叢編:Blood Advances
在線閱讀:http://www.sciencedirect.com/science/article/pii/S2473952920317997

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http://www.sciencedirect.com/science/article/pii/S2473952920317997

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