CRISPR Gene Therapy Enters Mainstream Medicine
The first CRISPR-based therapies have received regulatory approval. Casgevy treats sickle cell disease and beta-thalassemia by editing patients own blood stem cells. Results have been remarkable: patients who suffered frequent pain crises have been symptom-free for years. The cost exceeds 2 million dollars per patient, raising questions about access. Researchers are developing next-generation gene editing tools that are more precise and easier to deliver, potentially treating conditions from blindness to heart disease.