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

Autor: Gabr H; Clinical and Chemical Pathology, Faculty of Medicine, Cairo University, Cairo, Egypt., El Ghamrawy MK; Pediatric, Faculty of Medicine, Cairo University, Cairo, Egypt., Almaeen AH; Pathology Department, Jouf University, Sakakah, Saudi Arabia., Abdelhafiz AS; Clinical Pathology, National Cancer Institute, Cairo University, Cairo, Egypt., Hassan AOS; Clinical and Chemical Pathology, Faculty of Medicine, Cairo University, Cairo, Egypt., El Sissy MH; Clinical and Chemical Pathology, Faculty of Medicine, Cairo University, Cairo, Egypt. mahaelsissy@yahoo.com.
Jazyk: angličtina
Zdroj: Stem cell research & therapy [Stem Cell Res Ther] 2020 Sep 10; Vol. 11 (1), pp. 390. Date of Electronic Publication: 2020 Sep 10.
DOI: 10.1186/s13287-020-01876-4
Abstrakt: 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 cells in β-thalassemia with IVS-1-110 mutation.
Methods and Results: We successfully isolated CD34 + cells from peripheral blood of β-thalassemia patients with IVS-1-110 mutation. The cells were transfected with Cas9 endonuclease together with guide RNA to create double-strand breaks and knock out the mutation. The mutation-corrected CD34 + cells were subjected to erythroid differentiation by culturing in complete media containing erythropoietin.
Conclusion: CRISPR/Cas-9 is an effective tool for gene therapy that will broaden the spectrum of therapy and potentially improve the outcomes of β-thalassemia.
Databáze: MEDLINE
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