Induction of Fetal Hemoglobin by Introducing Natural Hereditary Persistence of Fetal Hemoglobin Mutations in the γ-Globin Gene Promoters for Genome Editing Therapies for β-Thalassemia

Reactivation of γ-globin expression is a promising therapeutic approach for β-hemoglobinopathies. Here, we propose a novel Cas9/AAV6-mediated genome editing strategy for the treatment of β-thalassemia: Natural HPFH mutations −113A > G, −114C > T, −117G>A, −175T > C, −195C > G, and −19...

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Detalhes bibliográficos

Principais autores:	Dian Lu, Zhiliang Xu, Zhiyong Peng, Yinghong Yang, Bing Song, Zeyu Xiong, Zhirui Ma, Hongmei Guan, Bangzhu Chen, Yukio Nakamura, Juan Zeng, Nengqing Liu, Xiaofang Sun, Diyu Chen
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Frontiers Media S.A. 2022-05-01
coleção:	Frontiers in Genetics
Assuntos:	fetal hemoglobin (HbF) hereditary persistence of fetal hemoglobin (HPFH) thalassemia genome editing adeno-associated virus (AAV)
Acesso em linha:	https://www.frontiersin.org/articles/10.3389/fgene.2022.881937/full
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