In a groundbreaking development in the field of gene editing, personalized CRISPR therapy is now being tested in clinical trials. This marks a significant advancement in the CRISPR revolution, as it involves specific gene editing tailored to individual patients. Previous FDA-approved CRISPR therapies targeted blood diseases like sickle cell anemia and thalassemia by removing bone marrow, editing the cells with CRISPR, and reinserting them. However, these edits were not patient-specific and targeted cells that could be replaced. The latest case report introduces two major breakthroughs – targeting an organ that cannot be removed and must be treated in vivo, and addressing a patient-specific mutation. This innovative approach holds promise for treating a wider range of genetic disorders and brings us closer to personalized medicine through gene editing.
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