Year: 2024 | Month: October-December | Volume: 9 | Issue: 4 | Pages: 55-62
DOI: https://doi.org/10.52403/gijhsr.20240404
Progress on CRISPR -Cas9 Gene Editing Technology in Sickle cell disease: A Review
Umar Bello Wada1, Shanti Nath2, Dr Mohammad Zeeshan3, Kushiram Sharma4, Yogesh Joshi5
1Department of Medical Laboratory Technology, 2Department of Medical Radiological Imaging,
Paramedical Sciences College, Mewar University, Gangrar, Rajasthan, India.
Corresponding Author: Umar Bello Wada
ABSTRACT
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas9 system as a method of Gene editing possess a broad range of applications for genetic modification, diagnosis and treatment for curing non-infectious (such as the Sickle cell disease) as well as infectious diseases. Initially identified in bacteria and archaea to fight plasmids' DNA and/or bacteriophage infecting bacteria. Cas9 and gRNA forms a complex to target and cleave the desired gene, providing defense (adaptive immunity) against viral infections to the host, also allow bacteria to recognize genetic sequences using specialized enzymes or CRISPR-associated proteins (Cas), including the DNA endonuclease, Cas9 systems (Molecular scissors). In this review we focus on the use of CRISPR/Cas9 gene-editing for curing Sickle cell disease (SCD), including the curative correction of SCD mutation in β-globin (HBB) and the induction of fetal hemoglobin to reverse sickling, that culminates with Casgevy and Lyfgenia Gene therapy approval by US Food and Drug Administration and UK Medicines and Healthcare Products Regulatory Agency. We summarize the major achievements and challenges, aiming to provide a clearer perspective on the potential of gene-editing based approaches in curing SCD using Prime editors to minimize off-target effect, cellular toxicity of delivery systems and durability as well as making it accessible and affordable to the low-income countries with highest prevalence in sickle cell disease.
Keywords: CRISPR -CAS9, Gene Editing Technology, Sickle cell disease, Cure affordability