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Gene therapy for metachromatic leukodystrophy

17 Sept 2016

Abstract excerpt

Leukodystrophies (LDs) are rare, often devastating genetic disorders with neurologic symptoms. There are currently no disease-specific therapeutic approaches for these diseases. In this review we use metachromatic leukodystrophy as an example to outline in the brief the therapeutic approaches to MLD that have been tested in animal models and in clinical trials, such as enzyme-replacement therapy, bone...

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PMID: 27638601
DOI: 10.1002/jnr.23792

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