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CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease

Genome editing is potentially a curative technique available to all individuals with β-hemoglobinopathies, including sickle cell disease (SCD). Fetal hemoglobin (HbF) inhibits sickle hemoglobin (HbS) polymerization, and it is well described that naturally occurring hereditary persistence of HbF (HPF...

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Detalles Bibliográficos
Autores principales:	Demirci, Selami, Leonard, Alexis, Essawi, Khaled, Tisdale, John F.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2021
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8526756/ https://www.ncbi.nlm.nih.gov/pubmed/34729375 http://dx.doi.org/10.1016/j.omtm.2021.09.010

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