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recipient mice resulted in sustained systemic levels of engineered GAA. Reduction of glycogen accumulation in muscle tissues and brain confirmed the superior activity of vIGF2-GAA compared to wild-type (WT) GAA, lowering urine glucose tetrasaccharide levels and correcting skeletal and cardiac phenotypes. These results demonstrate the therapeutic advantage of delivering engineered vIGF2-GAA and support the clinical translation of HSC gene therapy for PD.
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Wantuch et al. (2026) studied this question.