Author(s) :

Amna Qamar.

Volume/Issue :

Abstract :

Sickle cell disease is a genetic problem that occurs due to a change in the HBB gene, leading to misshapen red blood cells and faulty hemoglobin. Current treatments help control pain and complications but don't completely cure the disease. New developments in gene therapy, using techniques like CRISPR/Cas9 and lentiviral vectors, are offering a potential solution by fixing the problematic gene. A therapy called Casgevy, proven effective in clinical trials by reducing hospital visits and crisis episodes, is one example. However, challenges, such as high costs, scarce access in low-resource regions, and safety and efficacy concerns, must be addressed before widespread use. Still, gene therapy gives hope for a lasting cure for sickle cell disease, possibly improving the lives of affected individuals and revolutionizing genetic disease treatment.

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