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Smith, Barbara K; Martin, A Daniel; Lawson, Lee Ann et al. (2017) Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity. Exp Neurol 287:216-224
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Baligand, Celine; Todd, Adrian G; Lee-McMullen, Brittany et al. (2017) 13C/31P MRS Metabolic Biomarkers of Disease Progression and Response to AAV Delivery of hGAA in a Mouse Model of Pompe Disease. Mol Ther Methods Clin Dev 7:42-49
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Doerfler, Phillip A; Todd, Adrian G; Clément, Nathalie et al. (2016) Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease. Hum Gene Ther 27:43-59
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Doerfler, Phillip A; Nayak, Sushrusha; Corti, Manuela et al. (2016) Targeted approaches to induce immune tolerance for Pompe disease therapy. Mol Ther Methods Clin Dev 3:15053
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Drouin, Lauren M; Lins, Bridget; Janssen, Maria et al. (2016) Cryo-electron Microscopy Reconstruction and Stability Studies of the Wild Type and the R432A Variant of Adeno-associated Virus Type 2 Reveal that Capsid Structural Stability Is a Major Factor in Genome Packaging. J Virol 90:8542-51
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Conlon, Thomas J; Mah, Cathryn S; Pacak, Christina A et al. (2016) Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors. Hum Gene Ther Clin Dev 27:152-159
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Corti, Manuela; Smith, Barbara K; Falk, Darin J et al. (2015) Altered activation of the tibialis anterior in individuals with Pompe disease: Implications for motor unit dysfunction. Muscle Nerve 51:877-83
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Todd, Adrian G; McElroy, Jessica A; Grange, Robert W et al. (2015) Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease. Ann Neurol 78:222-34
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Falk, Darin J; Todd, Adrian Gary; Lee, Sooyeon et al. (2015) Peripheral nerve and neuromuscular junction pathology in Pompe disease. Hum Mol Genet 24:625-36
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Gruntman, Alisha M; Flotte, Terence R (2015) Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency. Hum Gene Ther Methods 26:77-81
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