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Clinical Trials for Gene Therapy in Lysosomal Diseases With CNS Involvement

16 Sept 2021

Abstract excerpt

There are over 70 known lysosomal storage disorders (LSDs), most caused by mutations in genes encoding lysosomal hydrolases. Central nervous system involvement is a hallmark of the majority of LSDs and, if present, generally determines the prognosis of the disease. Nonetheless, brain disease is currently poorly targeted by available therapies, including systemic enzyme replacement therapy, mostly (but not only)...

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PMID: 34604300
DOI: 10.3389/fmolb.2021.624988

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