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Page 1
In Situ Gene Therapy.
Santos HS, Rodrigues L, Vera LNP, Poletto E, Filippi-Chiela E, Dos Santos Bruschi LFR, Schuh R, Baldo G. Santos HS, et al. Among authors: vera lnp. Curr Gene Ther. 2021;21(5):406-430. doi: 10.2174/1566523221666210504103323. Curr Gene Ther. 2021. PMID: 33949931 Review.
Genome editing in lysosomal disorders.
Pimentel-Vera LN, Poletto E, Gonzalez EA, de Oliveira Poswar F, Giugliani R, Baldo G. Pimentel-Vera LN, et al. Prog Mol Biol Transl Sci. 2021;182:289-325. doi: 10.1016/bs.pmbts.2021.02.001. Epub 2021 Feb 22. Prog Mol Biol Transl Sci. 2021. PMID: 34175045
Identification of Ezetimibe and Pranlukast as Pharmacological Chaperones for the Treatment of the Rare Disease Mucopolysaccharidosis Type IVA.
Alméciga-Diaz CJ, Hidalgo OA, Olarte-Avellaneda S, Rodríguez-López A, Guzman E, Garzón R, Pimentel-Vera LN, Puentes-Tellez MA, Rojas-Rodriguez AF, Gorshkov K, Li R, Zheng W. Alméciga-Diaz CJ, et al. Among authors: pimentel vera ln. J Med Chem. 2019 Jul 11;62(13):6175-6189. doi: 10.1021/acs.jmedchem.9b00428. Epub 2019 Jun 25. J Med Chem. 2019. PMID: 31188588
Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice.
Schuh RS, Gonzalez EA, Tavares AMV, Seolin BG, Elias LS, Vera LNP, Kubaski F, Poletto E, Giugliani R, Teixeira HF, Matte U, Baldo G. Schuh RS, et al. Among authors: vera lnp. Gene Ther. 2020 Feb;27(1-2):74-84. doi: 10.1038/s41434-019-0113-4. Epub 2019 Dec 11. Gene Ther. 2020. PMID: 31827259