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Characterizing the mechanism of action for mRNA therapeutics for the treatment of propionic acidemia, methylmalonic acidemia, and phenylketonuria.
Baek R, Coughlan K, Jiang L, Liang M, Ci L, Singh H, Zhang H, Kaushal N, Rajlic IL, Van L, Dimen R, Cavedon A, Yin L, Rice L, Frassetto A, Guey L, Finn P, Martini PGV. Baek R, et al. Among authors: finn p. Nat Commun. 2024 May 7;15(1):3804. doi: 10.1038/s41467-024-47460-9. Nat Commun. 2024. PMID: 38714648 Free PMC article.
Altered transcriptomic immune responses of maintenance hemodialysis patients to the COVID-19 mRNA vaccine.
Chang YS, Huang K, Lee JM, Vagts CL, Ascoli C, Amin MR, Ghassemi M, Lora CM, Edafetanure-Ibeh R, Huang Y, Cherian RA, Sarup N, Warpecha SR, Hwang S, Goel R, Turturice BA, Schott C, Hernandez M, Chen Y, Jorgensen J, Wang W, Rasic M, Novak RM, Finn PW, Perkins DL. Chang YS, et al. Among authors: finn pw. Elife. 2024 Apr 24;13:e83641. doi: 10.7554/eLife.83641. Elife. 2024. PMID: 38656290 Free PMC article.
Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse model of Galactosemia.
Balakrishnan B, Yan X, McCue MD, Bellagamba O, Guo A, Winkler F, Thall J, Crawford L, Dimen R, Chen S, McEnaney S, Wu Y, Zimmer M, Sarkis J, Martini PGV, Finn PF, Lai K. Balakrishnan B, et al. Among authors: finn pf. Mol Ther Methods Clin Dev. 2024 Jan 17;32(1):101191. doi: 10.1016/j.omtm.2024.101191. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38352271 Free PMC article.
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria.
Gurung S, Timmermand OV, Perocheau D, Gil-Martinez AL, Minnion M, Touramanidou L, Fang S, Messina M, Khalil Y, Spiewak J, Barber AR, Edwards RS, Pinto PL, Finn PF, Cavedon A, Siddiqui S, Rice L, Martini PGV, Ridout D, Heywood W, Hargreaves I, Heales S, Mills PB, Waddington SN, Gissen P, Eaton S, Ryten M, Feelisch M, Frassetto A, Witney TH, Baruteau J. Gurung S, et al. Among authors: finn pf. Sci Transl Med. 2024 Jan 10;16(729):eadh1334. doi: 10.1126/scitranslmed.adh1334. Epub 2024 Jan 10. Sci Transl Med. 2024. PMID: 38198573 Free PMC article.
The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria.
Gurung S, Karamched S, Perocheau D, Seunarine KK, Baldwin T, Alrashidi H, Touramanidou L, Duff C, Elkhateeb N, Stepien KM, Sharma R, Morris A, Hartley T, Crowther L, Grunewald S, Cleary M, Mundy H, Chakrapani A, Batzios S, Davison J, Footitt E, Tuschl K, Lachmann R, Murphy E, Santra S, Uudelepp ML, Yeo M, Finn PF, Cavedon A, Siddiqui S, Rice L, Martini PGV, Frassetto A, Heales S, Mills PB, Gissen P, Clayden JD, Clark CA, Eaton S, Kalber TL, Baruteau J. Gurung S, et al. Among authors: finn pf. J Inherit Metab Dis. 2023 Dec 4. doi: 10.1002/jimd.12691. Online ahead of print. J Inherit Metab Dis. 2023. PMID: 38044746
Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice.
Anding A, Kinton S, Baranowski K, Brezzani A, De Busser H, Dufault MR, Finn P, Keefe K, Tetrault T, Li Y, Qiu W, Raes K, Vitse O, Zhang M, Ziegler R, Sardi SP, Hunter B, George K. Anding A, et al. Among authors: finn p. J Pharmacol Exp Ther. 2023 Nov;387(2):188-203. doi: 10.1124/jpet.123.001593. Epub 2023 Sep 7. J Pharmacol Exp Ther. 2023. PMID: 37679046
559 results