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Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice.

Mol Ther Methods Clin Dev · 2015 · PMC4445006 · PMID 26029718

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When compared to Gaa –/– , AAV9 appeared to reduce glycogen deposition in both the heart ( Figure 7i ) and diaphragm ( Figure 7j ) compared to Gaa –/– and ERT, however this did not reach statistical significance.

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