Induction of therapeutic levels of {HbF} in genome-edited primary β(0) 39-thalassaemia haematopoietic stem and progenitor cells
Articolo
Data di Pubblicazione:
2021
Citazione:
Induction of therapeutic levels of {HbF} in genome-edited primary β(0) 39-thalassaemia haematopoietic stem and progenitor cells / Mingoia, Maura; Caria Cristian, A.; Ye, Lin; Asunis, Isadora; Marongiu M., Franca; Manunza, Laura; Sollaino M., Carla; Wang, Jiaming; Cabriolu, Annalisa; Kurita, Ryo; Nakamura, Yukio; Cucca, Francesco; Kan Yuet, W.; Marini M., Giuseppina; Moi, Paolo. - In: BRITISH JOURNAL OF HAEMATOLOGY. - ISSN 1365-2141. - 192:2(2021), pp. 395-404. [10.1111/bjh.17167]
Abstract:
Hereditary persistence of fetal haemoglobin (HPFH) is the major modifier of the clinical severity of β-thalassaemia. The homozygous mutation c.-196 C{ extgreater}T in the Aγ-globin (HBG1) promoter, which causes Sardinian δβ(0) -thalassaemia, is able to completely rescue the β-major thalassaemia phenotype caused by the β(0)
Tipologia CRIS:
1.1 Articolo in rivista
Keywords:
CRISPR/Cas9, fetal hemoglobin induction, genome editing, sardinian δβ0-thalassemia, β0-thalassemia
Elenco autori:
Mingoia, Maura; Caria Cristian, A.; Ye, Lin; Asunis, Isadora; Marongiu M., Franca; Manunza, Laura; Sollaino M., Carla; Wang, Jiaming; Cabriolu, Annalisa; Kurita, Ryo; Nakamura, Yukio; Cucca, Francesco; Kan Yuet, W.; Marini M., Giuseppina; Moi, Paolo
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