Preclinical assessment of GNE wt /bi-shRNA-GNE M743T lipoplex product development for GNE myopathy

20260 citationsJournal Articlegold Open Access

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These results encourage future studies, potentially leading toward clinical testing of GNEwt/bi-shRNA-GNEM743T lipoplex for GNE myopathy.

Published in: Future Science OA

Volume 12, Issue 1, pp. 2635722-2635722

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