• Neerja Masih
• Arundhati Rej
• Madhurima Tiwari

Sickle cell Anemia is a severe inherited blood disorder that develops due to a mutation in the beta chain protein of hemoglobin, a crucial protein in red blood cells responsible for carrying oxygen from the lungs to all parts of the body. This genetic alteration causes the red blood cells to assume an abnormal sickle or crescent shape, which can lead to blockages in the blood vessels. The blocked vessels can limit the amount of oxygen reaching the body tissues, causing intense pain and other complications such as organ damage, infections, and stroke. The current treatment options for sickle cell disease are limited and pose significant risks. However, gene therapy, specifically CRISPR-Cas9 technology, has emerged as a promising new treatment avenue for this condition. This innovative approach involves editing the DNA to correct the mutation that causes sickle cell anemia. This correction can potentially lead to healthy red blood cells that can carry oxygen efficiently and reduce the risk of complications. CRISPR-Cas9 is a revolutionary gene-editing technology that can accurately target and modify specific base pairs in DNA. Researchers use a guide RNA molecule to direct the Cas9 protein to the specific location in the DNA sequence where the mutation causing sickle cell disease occurs. The Cas9 protein then cuts the DNA, and the cell's repair mechanisms replace the mutated DNA with the correct version, fixing the genetic defect. Moreover, CRISPR-Cas9 technology demonstrates the potential to treat various other genetic disorders beyond sickle cell disease. The technology is still undergoing research and development, and more investigations are necessary to determine its safety and efficacy for treating sickle cell disease and other genetic conditions. In this review, we will delve into the details of treating sickle cell anemia through the application of CRISPR-Cas9.

Gene therapy; Hematopoietic stem cell transplantation; Sickle cell Diseases; Gene delivery; CRISPR CAS 9; Sickle cell anemia .

"CRISPR CAS 9 AS A POTENTIAL THERAPY AGAINST SICKLE CELL ANEMIA", International Journal of Emerging Technologies and Innovative Research (www.jetir.org), ISSN:2349-5162, Vol.11, Issue 10, page no.f315-f321, October-2024, Available :http://www.jetir.org/papers/JETIR2410527.pdf

"CRISPR CAS 9 AS A POTENTIAL THERAPY AGAINST SICKLE CELL ANEMIA", International Journal of Emerging Technologies and Innovative Research (www.jetir.org | UGC and issn Approved), ISSN:2349-5162, Vol.11, Issue 10, page no. ppf315-f321, October-2024, Available at : http://www.jetir.org/papers/JETIR2410527.pdf