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AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice.
Doyle BM, Turner SMF, Sunshine MD, Doerfler PA, Poirier AE, Vaught LA, Jorgensen ML, Falk DJ, Byrne BJ, Fuller DD. Doyle BM, et al. Among authors: falk dj. Mol Ther Methods Clin Dev. 2019 Aug 31;15:194-203. doi: 10.1016/j.omtm.2019.08.009. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31660421 Free PMC article.
Pompe disease gene therapy.
Byrne BJ, Falk DJ, Pacak CA, Nayak S, Herzog RW, Elder ME, Collins SW, Conlon TJ, Clement N, Cleaver BD, Cloutier DA, Porvasnik SL, Islam S, Elmallah MK, Martin A, Smith BK, Fuller DD, Lawson LA, Mah CS. Byrne BJ, et al. Among authors: falk dj. Hum Mol Genet. 2011 Apr 15;20(R1):R61-8. doi: 10.1093/hmg/ddr174. Epub 2011 Apr 25. Hum Mol Genet. 2011. PMID: 21518733 Free PMC article. Review.
Hypoglossal neuropathology and respiratory activity in pompe mice.
Lee KZ, Qiu K, Sandhu MS, Elmallah MK, Falk DJ, Lane MA, Reier PJ, Byrne BJ, Fuller DD. Lee KZ, et al. Among authors: falk dj. Front Physiol. 2011 Jun 30;2:31. doi: 10.3389/fphys.2011.00031. eCollection 2011. Front Physiol. 2011. PMID: 21747768 Free PMC article.
55 results