A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition

Elizabeth A. Traxler; Yu Yao; Yong‐Dong Wang; Kaitly J. Woodard; Ryo Kurita; Yukio Nakamura; Jim R. Hughes; Ross C. Hardison; Gerd A. Blobel; Chunliang Li; Mitchell J Weiss

doi:10.1038/nm.4170

A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition

Elizabeth A. Traxler(St. Jude Children's Research Hospital), Yu Yao(St. Jude Children's Research Hospital), Yong‐Dong Wang(St. Jude Children's Research Hospital), Kaitly J. Woodard(St. Jude Children's Research Hospital), Ryo Kurita(RIKEN BioResource Research Center), Yukio Nakamura(University of Tsukuba), Jim R. Hughes(University of Oxford), Ross C. Hardison(Pennsylvania State University), Gerd A. Blobel(Children's Hospital of Philadelphia), Chunliang Li(St. Jude Children's Research Hospital), Mitchell J Weiss(St. Jude Children's Research Hospital)

Nature Medicine

August 15, 2016

10.1038/nm.4170

Cited by 385Open Access

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