Waking up the silenced beauty: CRISPR/Cas9 mediated reactivation of fetal hemoglobin genes to treat severe beta-thalassemia in young patients

Liren Wang , Stefan Siwko , Dali Li

Life Medicine ›› 2023, Vol. 2 ›› Issue (2) : 4

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Life Medicine ›› 2023, Vol. 2 ›› Issue (2) : 4 DOI: 10.1093/lifemedi/lnad009
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Waking up the silenced beauty: CRISPR/Cas9 mediated reactivation of fetal hemoglobin genes to treat severe beta-thalassemia in young patients

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Liren Wang, Stefan Siwko, Dali Li. Waking up the silenced beauty: CRISPR/Cas9 mediated reactivation of fetal hemoglobin genes to treat severe beta-thalassemia in young patients. Life Medicine, 2023, 2(2): 4 DOI:10.1093/lifemedi/lnad009

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References

[1]

Frangoul H, Altshuler D, Cappellini MD, et al. CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia. New Engl J Med 2020;384:252–60.

[2]

Fu B, Liao J, Chen S, et al. CRISPR–Cas9-mediated gene editing of the BCL11A enhancer for pediatric β0/β0 transfusion-dependent β-thalassemia. Nat Med 2022;28:1573–80.

[3]

Wang L, Li L, Ma Y, et al. Reactivation of γ-globin expression through Cas9 or base editor to treat β-hemoglobinopathies. Cell Res 2020;30:276–8.

[4]

Newby GA, Yen JS, Woodard KJ, et al. Base editing of haematopoietic stem cells rescues sickle cell disease in mice. Nature 2021;595:295–302.

[5]

Dever DP, Bak RO, Reinisch A, et al. CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells. Nature 2016;539:384–9.

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The Author(s) 2023. Published by Oxford University Press on behalf of Higher Education Press.

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