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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...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2020
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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 |