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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: kabbej n. Hum Mol Genet. 2023 Jan 13;32(3):417-430. doi: 10.1093/hmg/ddac209. Hum Mol Genet. 2023. PMID: 35997776 Free PMC article.