GENOMIC EDITING: NEW HOPE IN THE TREATMENT OF SICKLE CELL DISEASE?
DOI:
https://doi.org/10.47820/recima21.v5i6.5328Keywords:
Anemia; , Edition, SicklingAbstract
Sickle cell disease (SCD) is a group of hereditary hemoglobinopathies characterized by mutations that affect the β-globin chain of hemoglobin. Objective: to group what already exists in the literature on the use of the CRISPR-Cas9 system in the treatment of sickle cell disease. Materials and methods: This is an integrative review, in which the guiding question was “Is the CRISPR-Cas9 system capable of treating sickle cell disease?”. The search for articles was carried out in PubMed using the terms “CRISPR-cas9”, “sickle cell”, “anemia” combined with Boolean operators. Results and Discussion: Correction of the disease causing the sickle cell mutation using gene editing represents the most direct therapeutic approach. The β-globin-targeted pre-complexed CRISPR gRNA/Cas9 ribonucleoprotein complex along with the DNA donor template are delivered into hematopoietic stem cells and autologous progenitors isolated from patients with sickle cell disease, resulting in repair mediated by homology-directed repair of the causative mutation. CRISPR-Cas9-mediated genetic modification has demonstrated variable efficiency, specificity, and persistence in hematopoietic stem cells. Conclusion: The recent discovery of CRISPR/Cas9 not only revolutionized genome engineering, but also brought the possibility of translating these concepts into a clinically meaningful reality.
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