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Article

Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease

25 Jan 2021

Abstract excerpt

Neonatal AAV9-gene therapy of the lysosomal enzyme galactosylceramidase (GALC) significantly ameliorates central and peripheral neuropathology, prolongs survival, and largely normalizes motor deficits in Twitcher mice. Despite these therapeutic milestones, new observations identified the presence of multiple small focal demyelinating areas in the brain after 6-8 months. These lesions are in stark contrast to the...

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PMID: 33508430
DOI: 10.1016/j.ymthe.2021.01.026

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Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe diseasePMID 33508430Read ↗

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