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Page 1
In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions.
Nat Commun. 2022 Aug 25;13(1):5012. doi: 10.1038/s41467-022-32576-7.
Nat Commun. 2022.
PMID: 36008405
Free PMC article.
Development of a porcine model of phenylketonuria with a humanized R408W mutation for gene editing.
Kaiser RA, Carlson DF, Allen KL, Webster DA, VanLith CJ, Nicolas CT, Hillin LG, Yu Y, Kaiser CW, Wahoff WR, Hickey RD, Watson AL, Winn SR, Thöny B, Kern DR, Harding CO, Lillegard JB.
Kaiser RA, et al. Among authors: hillin lg.
PLoS One. 2021 Jan 25;16(1):e0245831. doi: 10.1371/journal.pone.0245831. eCollection 2021.
PLoS One. 2021.
PMID: 33493163
Free PMC article.
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Use of an adeno-associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model.
Kaiser RA, Weber ND, Trigueros-Motos L, Allen KL, Martinez M, Cao W, VanLith CJ, Hillin LG, Douar A, González-Aseguinolaza G, Aldabe R, Lillegard JB.
Kaiser RA, et al. Among authors: hillin lg.
J Inherit Metab Dis. 2021 Nov;44(6):1369-1381. doi: 10.1002/jimd.12392. Epub 2021 Aug 19.
J Inherit Metab Dis. 2021.
PMID: 33896013
Free PMC article.
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