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Lysosomal storage disease: Gene therapy on both sides of the blood–brain barrier

9 Oct 2014

Abstract excerpt

Most lysosomal storage disorders affect the nervous system as well as other tissues and organs of the body. Previously, the complexities of these diseases, particularly in treating neurologic abnormalities, were too great to surmount. However, based on recent developments there are realistic expectations that effective therapies are coming soon. Gene therapy offers the possibility of affordable, comprehensive...

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PMID: 25410058
DOI: 10.1016/j.ymgme.2014.09.011

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