Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease

Autologous hematopoietic stem cell (HSC)-targeted gene therapy provides a one-time cure for various genetic diseases including sickle cell disease (SCD) and β-thalassemia. SCD is caused by a point mutation (20A > T) in the β-globin gene. Since SCD is the most common single-gene disorder, curing S...

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Detalles Bibliográficos
Autores principales: Germino-Watnick, Paula, Hinds, Malikiya, Le, Anh, Chu, Rebecca, Liu, Xiong, Uchida, Naoya
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9180595/
https://www.ncbi.nlm.nih.gov/pubmed/35681538
http://dx.doi.org/10.3390/cells11111843

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9180595/
https://www.ncbi.nlm.nih.gov/pubmed/35681538
http://dx.doi.org/10.3390/cells11111843

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