CRISPR-Cas9 continues to be a promising treatment method for many diseases, with a few even already being improved by the FDA for treatment, such as Casgevy. It was approved by the FDA in December 2023 for people aged 12 and older with recurring vaso-oclusive pain crises, and then approved for people within the samge age range with Sickle Cell Disease and Beta Thalassemia in January 24. Casgevy is a CRISPR based gene editing therapy that shows a lot of promise for upcomed therapies of this sort and respresents a major breakthrough in genetic engineering. It targets a specific gene known as the BCl11a gene by limiting it's expression, rather than by directly targeting the main DNA sequence of the disease itself.The goal of this site is to explore this treatment, what parties are involves, the various clinical trials the treatment went through, and what lessons it brings for successfull gene therapy treatments in the near future.
Significance of the BCL11a Gene Characteristics of the SpCas9 Nuclease