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Article

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

Stem cell research & therapy - 10 Sept 2020

Gabr Hala, El Ghamrawy Mona Kamal, Almaeen Abdulrahman H, Abdelhafiz Ahmed Samir, Hassan Aya Osama Saad, El Sissy Maha Hamdi

Abstract excerpt

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 these disorders. The study aimed to investigate the utility of CRISPR-CAS9 for gene modification of hematopoietic stem...

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Identifiers

PMID: 32912325
DOI: 10.1186/s13287-020-01876-4
PMCID: PMC7488347

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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