alexa Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors.
Nephrology

Nephrology

Journal of Nephrology & Therapeutics

Author(s): Mah C, Pacak CA, Cresawn KO, Deruisseau LR, Germain S, , Mah C, Pacak CA, Cresawn KO, Deruisseau LR, Germain S,

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Abstract Pompe disease is caused by a lack of functional lysosomal acid alpha-glucosidase (GAA) and can ultimately lead to fatal hypertrophic cardiomyopathy and respiratory insufficiency. Previously, we demonstrated the ability of recombinant adeno-associated virus serotype 1 (rAAV2/1) vector to restore the therapeutic levels of cardiac and diaphragmatic GAA enzymatic activity in vivo in a mouse model of Pompe disease. We have further characterized cardiac and respiratory function in rAAV2/1-treated animals 1 year post-treatment. Similar to the patient population, electrocardiogram measurements (P-R interval) are significantly shortened in the Pompe mouse model. In rAAV2/1-treated mice, we show a significant improvement in cardiac conductance with prolonged P-R intervals of 39.34+/-1.6 ms, as compared to untreated controls (35.58+/-0.57 ms) (P
  • DOI: 10.1038/sj.mt.6300100
  • This article was published in Mol Ther and referenced in Journal of Nephrology & Therapeutics

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