Researchers from Children's Hospital of Philadelphia (CHOP) have tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that affects the brain, lungs, skin, and skeleton with no currently approved treatments.
Researchers report encouraging first evidence of effective new gene therapy to treat multiple sulfatase deficiency retrieved 9 September 2024 from https://medicalxpress.com/news/2024-09-evidence-effective-gene-therapy-multiple.html
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