RISPR Gene Therapy Mechanism Discovered

St. Jude researchers identified how CRISPR-based gene therapy treats sickle cell disease and β-thalassemia by disrupting three-dimensional genome structure. Published in November 2025, the study revealed previously unknown treatment opportunities for these blood disorders. The findings explain the molecular mechanism behind the first FDA-approved CRISPR therapy and may lead to improved protocols for genetic diseases, representing a significant advance in precision medicine and genomic editing applications https://www.stjude.org/media-resources/news-releases/2025-medicine-science-news.html