The treatment will help those suffering from the most severe form of sickle cell disease (SCD), an illness that disproportionately affects people of African, Middle Eastern and Indian descent.
On Friday the Food and Drug Administration (FDA) approved the first gene-editing treatment for human illness in a landmark decision. The treatment will help those suffering from the most severe form of sickle cell disease (SCD), an illness that disproportionately affects people of African, Middle Eastern and Indian descent.»Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.»The treatment, called Casgevy, comes from the Boston-based companies Vertex Pharmaceuticals and CRISPR Therapeutics. CRISPR (which stands for clustered regularly interspaced short palindromic repeats) is a form of technology that allows research scientists to selectively modify the DNA of living organisms, according to the National Institutes of Health (NIH).
