Nutritional and Metabolic Diseases: News and Research on Multiple Sulfatase Deficiency Disease

MeSH tree: C18.452.132.100.435.825.850.750

Gene therapy and bone marrow transplant alleviate ultra-rare genetic disease symptoms

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and bone marrow transplantation—to alleviate symptoms of multiple sulfatase deficiency (MSD), an ultra-rare ...

Jan 29, 2025

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Researchers report encouraging first evidence of effective new gene therapy to treat multiple sulfatase deficiency

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, ...

Sep 9, 2024

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