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)

Induction of therapeutic levels of {HbF} in genome-edited primary β(0) 39-thalassaemia haematopoietic stem and progenitor cells / Mingoia, M., Caria Cristian, A., Ye, L., Asunis, I., Marongiu M., F., Manunza, L., Sollaino M., C., Wang, J., Cabriolu, A., Kurita, R., Nakamura, Y., Cucca, F., Kan Yuet, W., Marini M., G., Moi, P.. - In: BRITISH JOURNAL OF HAEMATOLOGY. - ISSN 1365-2141. - 192:2(2021), pp. 395-404. [10.1111/bjh.17167]

Induction of therapeutic levels of {HbF} in genome-edited primary β(0) 39-thalassaemia haematopoietic stem and progenitor cells

Cucca Francesco;Moi Paolo
2021-01-01

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)
2021
192
2
395
404
10
CRISPR/Cas9, fetal hemoglobin induction, genome editing, sardinian δβ0-thalassemia, β0-thalassemia
Mingoia, Maura; Caria Cristian, A.; Ye, Lin; Asunis, Isadora; Marongiu M., Franca; Manunza, Laura; Sollaino M., Carla; Wang, Jiaming; Cabriolu, Annali...espandi
Induction of therapeutic levels of {HbF} in genome-edited primary β(0) 39-thalassaemia haematopoietic stem and progenitor cells / Mingoia, M., Caria Cristian, A., Ye, L., Asunis, I., Marongiu M., F., Manunza, L., Sollaino M., C., Wang, J., Cabriolu, A., Kurita, R., Nakamura, Y., Cucca, F., Kan Yuet, W., Marini M., G., Moi, P.. - In: BRITISH JOURNAL OF HAEMATOLOGY. - ISSN 1365-2141. - 192:2(2021), pp. 395-404. [10.1111/bjh.17167]
info:eu-repo/semantics/article
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11388/245758
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