Genome editing for sickle cell disease: still time to correct?
Sickle cell disease (SCD) is an inherited blood disorder, due to a single point mutation in the β-globin gene (HBB) leading to multisystemic manifestations and it affects millions of people worldwide. The monogenic nature of the disease and the availability of autologous hematopoietic stem cells (HS...
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Format: | Book |
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Frontiers Media S.A.,
2023-11-01T00:00:00Z.
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A1234.567 |
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