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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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Detalles Bibliográficos
Autores principales:	Ceglie, Giulia, Lecis, Marco, Canciani, Gabriele, Algeri, Mattia, Frati, Giacomo
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2023
Materias:	Pediatrics
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10652763/ https://www.ncbi.nlm.nih.gov/pubmed/38027257 http://dx.doi.org/10.3389/fped.2023.1249275

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