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ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia.
Ilyinskii PO, Michaud AM, Rizzo GL, Roy CJ, Leung SS, Elkins SL, Capela T, Chowdhury A, Li L, Chandler RJ, Manoli I, Andres-Mateos E, Johnston LPM, Vandenberghe LH, Venditti CP, Kishimoto TK. Ilyinskii PO, et al. Among authors: vandenberghe lh. Mol Ther Methods Clin Dev. 2021 Jul 16;22:279-292. doi: 10.1016/j.omtm.2021.06.015. eCollection 2021 Sep 10. Mol Ther Methods Clin Dev. 2021. PMID: 34485611 Free PMC article.
AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations.
Pacouret S, Bouzelha M, Shelke R, Andres-Mateos E, Xiao R, Maurer A, Mevel M, Turunen H, Barungi T, Penaud-Budloo M, Broucque F, Blouin V, Moullier P, Ayuso E, Vandenberghe LH. Pacouret S, et al. Among authors: vandenberghe lh. Mol Ther. 2017 Jun 7;25(6):1375-1386. doi: 10.1016/j.ymthe.2017.04.001. Epub 2017 Apr 17. Mol Ther. 2017. PMID: 28427840 Free PMC article.
Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65.
Hudry E, Andres-Mateos E, Lerner EP, Volak A, Cohen O, Hyman BT, Maguire CA, Vandenberghe LH. Hudry E, et al. Among authors: vandenberghe lh. Mol Ther Methods Clin Dev. 2018 Jul 23;10:197-209. doi: 10.1016/j.omtm.2018.07.006. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30109242 Free PMC article.
FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia.
Manoli I, Sysol JR, Epping MW, Li L, Wang C, Sloan JL, Pass A, Gagné J, Ktena YP, Li L, Trivedi NS, Ouattara B, Zerfas PM, Hoffmann V, Abu-Asab M, Tsokos MG, Kleiner DE, Garone C, Cusmano-Ozog K, Enns GM, Vernon HJ, Andersson HC, Grunewald S, Elkahloun AG, Girard CL, Schnermann J, DiMauro S, Andres-Mateos E, Vandenberghe LH, Chandler RJ, Venditti CP. Manoli I, et al. Among authors: vandenberghe lh. JCI Insight. 2018 Dec 6;3(23):e124351. doi: 10.1172/jci.insight.124351. JCI Insight. 2018. PMID: 30518688 Free PMC article.
Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries.
Schmit PF, Pacouret S, Zinn E, Telford E, Nicolaou F, Broucque F, Andres-Mateos E, Xiao R, Penaud-Budloo M, Bouzelha M, Jaulin N, Adjali O, Ayuso E, Vandenberghe LH. Schmit PF, et al. Among authors: vandenberghe lh. Mol Ther Methods Clin Dev. 2019 Nov 26;17:107-121. doi: 10.1016/j.omtm.2019.11.014. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2019. PMID: 31909084 Free PMC article.
110 results