The past, present, and future of gene therapy From early experiments in rare genetic diseases to next-generation tools that can edit, insert, and regulate genes, gene therapy is transforming what’s possible for patients.

https://www.drugdiscoverynews.com/the-past-present-and-future-of-gene-therapy-17093 When Connie Elson was diagnosed with metachromatic leukodystrophy (MLD) at the age of five, her family had never heard of the disease. A rare inherited neurodegenerative disorder, MLD is caused by mutations in the ARSA (arylsulfatase A) gene, leading to a toxic build-up of sulfatides that progressively destroy the nervous system.