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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Detalles Bibliográficos
Autores principales: Lu, Dian, Xu, Zhiliang, Peng, Zhiyong, Yang, Yinghong, Song, Bing, Xiong, Zeyu, Ma, Zhirui, Guan, Hongmei, Chen, Bangzhu, Nakamura, Yukio, Zeng, Juan, Liu, Nengqing, Sun, Xiaofang, Chen, Diyu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Genetics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9152165/
https://www.ncbi.nlm.nih.gov/pubmed/35656314
http://dx.doi.org/10.3389/fgene.2022.881937

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