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Human Gene Therapy|April 11, 2013
Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomesBarbara K Smith, Shelley W Collins, Thomas J Conlon, et al.
Proceedings of the National Academy of Sciences of the United States of America|May 31, 2008
Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower dosesLi Zhong, Baozheng Li, Cathryn S Mah, et al.
Journal of Biomedicine & Biotechnology|February 15, 2011
Glycogen storage disease type Ia in canines: a model for human metabolic and genetic liver diseaseAndrew Specht, Laurie Fiske, Kirsten Erger, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 28, 2010
Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established diseaseCathryn S Mah, Darin J Falk, Sean A Germain, et al.
Ophthalmology and Therapy|July 10, 2025
Real-World Experience with Lifitegrast Ophthalmic Solution in Patients with Dry Eye Disease: A Provider Survey in the USA and CanadaCecelia Koetting, Justin Schweitzer, Kelly K Nichols, et al.
AIDS (London, England)|June 21, 2021
Attributing health benefits to preventing HIV infections versus improving health outcomes among people living with HIV: an analysis in six US citiesEmanuel Krebs, Eva Enns, Xiao Zang, et al.
American Journal of Human Genetics|May 29, 2004
Age-related maculopathy: a genomewide scan with continued evidence of susceptibility loci within the 1q31, 10q26, and 17q25 regionsDaniel E Weeks, Yvette P Conley, Hui-Ju Tsai, et al.
Plos One|February 19, 2021
Vitamin D supplementation among Bangladeshi children under-five years of age hospitalised for severe pneumonia: A randomised placebo controlled trialFahmida Chowdhury, Abu Sadat Mohammad Sayeem Bin Shahid, Mosharrat Tabassum, et al.
Human Molecular Genetics|April 27, 2011
Pompe disease gene therapyBarry J Byrne, Darin J Falk, Christina A Pacak, et al.
Human Gene Therapy|February 19, 2010
Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type IaDavid A Weinstein, Catherine E Correia, Thomas Conlon, et al.
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