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mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease.
Cao J, Choi M, Guadagnin E, Soty M, Silva M, Verzieux V, Weisser E, Markel A, Zhuo J, Liang S, Yin L, Frassetto A, Graham AR, Burke K, Ketova T, Mihai C, Zalinger Z, Levy B, Besin G, Wolfrom M, Tran B, Tunkey C, Owen E, Sarkis J, Dousis A, Presnyak V, Pepin C, Zheng W, Ci L, Hard M, Miracco E, Rice L, Nguyen V, Zimmer M, Rajarajacholan U, Finn PF, Mithieux G, Rajas F, Martini PGV, Giangrande PH. Cao J, et al. Among authors: graham ar. Nat Commun. 2021 May 25;12(1):3090. doi: 10.1038/s41467-021-23318-2. Nat Commun. 2021. PMID: 34035281 Free PMC article.
A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-associated Virus Serotypes Upon Cerebrospinal Fluid Administration in Pigs.
Sorrentino NC, Maffia V, Strollo S, Cacace V, Romagnoli N, Manfredi A, Ventrella D, Dondi F, Barone F, Giunti M, Graham AR, Huang Y, Kalled SL, Auricchio A, Bacci ML, Surace EM, Fraldi A. Sorrentino NC, et al. Among authors: graham ar. Mol Ther. 2016 Feb;24(2):276-286. doi: 10.1038/mt.2015.212. Epub 2015 Dec 7. Mol Ther. 2016. PMID: 26639405 Free PMC article.
Sustained Systemic Glucocerebrosidase Inhibition Induces Brain α-Synuclein Aggregation, Microglia and Complement C1q Activation in Mice.
Rocha EM, Smith GA, Park E, Cao H, Graham AR, Brown E, McLean JR, Hayes MA, Beagan J, Izen SC, Perez-Torres E, Hallett PJ, Isacson O. Rocha EM, et al. Among authors: graham ar. Antioxid Redox Signal. 2015 Aug 20;23(6):550-64. doi: 10.1089/ars.2015.6307. Epub 2015 Jul 29. Antioxid Redox Signal. 2015. PMID: 26094487 Free PMC article.