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Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy.
Mol Ther. 2024 Jan 3;32(1):44-58. doi: 10.1016/j.ymthe.2023.11.014. Epub 2023 Nov 11.
Mol Ther. 2024.
PMID: 37952085
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: herbst z.
Hum Mol Genet. 2023 Jan 13;32(3):417-430. doi: 10.1093/hmg/ddac209.
Hum Mol Genet. 2023.
PMID: 35997776
Free PMC article.
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