Abstract: Background: Monogenic disorders are inherited diseases that are mainly caused by changes in a single gene. Lower the disease burden but they usually do not fix the original genetic problem. CRISPR‑Cas9 genome editing offers a long‑lasting therapy idea by changing DNA or its control routes.
Objective: This systematic review checked how well CRISPR‑Cas9 therapies work how safe CRISPR‑Cas9 editing is, how well CRISPR‑Cas9 can be delivered and how ready CRISPR‑Cas9 treatments are for real‑world use in disorders.
Methods: We looked at papers from 2016 to 2025 in PubMed/MEDLINE, Embase, Scopus, Web of Science and Google Scholar. We pulled data on CRISPR‑Cas9 efficacy, CRISPR‑Cas9 editing efficiency, CRISPR‑Cas9 safety, CRISPR‑Cas9 delivery, CRISPR‑Cas9 durability and CRISPR‑Cas9 clinical translation. We used the PRISMA method for study selection. Of 300 records, we found 25 studies fit the criteria.
Results: CRISPR‑Cas9 showed hopeful therapy results in blood, metabolism, liver, muscle and eye disorders. The best evidence was for sickle cell disease and transfusion‑dependent β‑thalassemia, where exagamglogene autotemcel (exa‑cel; Casgevy) gave benefits. In conclusion, CRISPR‑Cas9 has moved from a lab tool to a proven therapy platform for monogenic disorders.
Keywords: CRISPR‑Cas9, Sickle cell disease, DNA damage, monogenic disorder, Inclusion/Exclusion criterion, PRISMA.
Title: CRISPR- Cas9 for the Treatment of Monogenic Disorders: A Systematic Review of Therapeutic Efficacy, Genome - Editing Safety, Delivery and Translational Feasibility
Author: Krupa Shah, Keya Shah
International Journal of Interdisciplinary Research and Innovations
ISSN 2348-1218 (print), ISSN 2348-1226 (online)
Vol. 14, Issue 4, October 2026 - December 2026
Page No: 14-20
Research Publish Journals
Website: www.researchpublish.com
Published Date: 27-October-2026