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Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector.
Rouse CJ, Hawkins K, Kabbej N, Dalugdug J, Kunta A, Kim MJ, Someya S, Herbst Z, Gelb M, Dinelli I, Butterworth E, Falk DJ, Rosenkrantz E, Elmohd H, Khaledi H, Mowafy S, Ashby F, Heldermon CD. Rouse CJ, et al. Among authors: falk dj. Hum Mol Genet. 2023 Jan 13;32(3):417-430. doi: 10.1093/hmg/ddac209. Hum Mol Genet. 2023. PMID: 35997776 Free PMC article.
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.
52 results