Why the study?
GNE myopathy has no available therapy, leading to the development of a muscle-specific GNE construct packaged with AAVrh74 for efficacy evaluation in an animal model.
Does systemic delivery of rAAVrh74.MCK.GNE viral vector improve muscle function and GNE expression in a Gne-/- hGNED207V Tg mouse model of GNE Myopathy?
Does systemic delivery of rAAVrh74.MCK.GNE viral vector improve muscle function and GNE expression in a Gne-/- hGNED207V Tg mouse model of GNE Myopathy?
The rAAVrh74.MCK.GNE vector successfully expresses human wt GNE in murine muscles, but efficacy assessment is limited by the lack of a reliable animal model exhibiting GNE myopathy characteristics.
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Vector achieves long-term GNE expression in murine muscle; leaves open therapeutic efficacy in GNE myopathy due to inadequate disease model.
A 2021 study studied this question.
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