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CRISPR-mediated gene modification of hematopoietic stem cells with beta-thalassemia IVS-1-110 mutation

BACKGROUND: β-Thalassemias represent a group of genetic disorders caused by human hemoglobin beta (HBB) gene mutations. The radical curative approach is to correct the mutations causing the disease. CRISPR-CAS9 is a novel gene-editing technology that can be used auspiciously for the treatment of the...

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Detalles Bibliográficos
Autores principales: Gabr, Hala, El Ghamrawy, Mona Kamal, Almaeen, Abdulrahman H., Abdelhafiz, Ahmed Samir, Hassan, Aya Osama Saad, El Sissy, Maha Hamdi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7488347/
https://www.ncbi.nlm.nih.gov/pubmed/32912325
http://dx.doi.org/10.1186/s13287-020-01876-4