HIGHLIGHTS
- who: Linda Rossini from the Pediatric Hematology, Oncology and Stem Cell Transplant Division, Padua University Hospital, Padua, Italy, Fondazione have published the paper: New Indications for Hematopoietic Stem Cell Gene Therapy in Lysosomal Storage Disorders, in the Journal: (JOURNAL)
- what: This approach showed better correction of memory deficits, inflammation, and HS storage in the brain as compared to a LV encoding only IDS . Recent advances in HSC GT for patients with LSDs has shown that this approach provides a cure for previously untreatable conditions such as MLD and might be efficacious also in other indications . . .
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