HBG1/2和BCL11A作为治疗β-血红蛋白病的基因组编辑靶点的非临床评价 - Suppr

Nonclinical evaluation of HBG1/2 and BCL11A as genome-editing targets for the treatment of β-hemoglobinopathies.

作者信息

Janoudi Tusneem, Jagdale Mrunali, Wu Meng, Gorlla Sanjana, Zhang Peisheng, Shao Ying, Li Li, Bowley Sheryl R, Marco Eugenio, Chang Kai-Hsin

机构信息

Editas Medicine Inc, Cambridge, MA.

出版信息

Blood Adv. 2025 Feb 25;9(4):808-813. doi: 10.1182/bloodadvances.2024014040.

DOI:10.1182/bloodadvances.2024014040

PMID:39637306

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11869951/

Abstract

摘要

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e948/11869951/eedce1416302/BLOODA_ADV-2024-014040-ga1.jpg

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Nonclinical evaluation of HBG1/2 and BCL11A as genome-editing targets for the treatment of β-hemoglobinopathies.

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