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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: chandler rj. 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.
Genetic and genomic systems to study methylmalonic acidemia.
Chandler RJ, Venditti CP. Chandler RJ, et al. Mol Genet Metab. 2005 Sep-Oct;86(1-2):34-43. doi: 10.1016/j.ymgme.2005.07.020. Epub 2005 Sep 22. Mol Genet Metab. 2005. PMID: 16182581 Free PMC article. Review.
Mitochondrial dysfunction in mut methylmalonic acidemia.
Chandler RJ, Zerfas PM, Shanske S, Sloan J, Hoffmann V, DiMauro S, Venditti CP. Chandler RJ, et al. FASEB J. 2009 Apr;23(4):1252-61. doi: 10.1096/fj.08-121848. Epub 2008 Dec 16. FASEB J. 2009. PMID: 19088183 Free PMC article.
53 results