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Items: 11

1.

Genome Editing for Mucopolysaccharidoses.

Poletto E, Baldo G, Gomez-Ospina N.

Int J Mol Sci. 2020 Jan 13;21(2). pii: E500. doi: 10.3390/ijms21020500. Review.

2.

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.

Gene Ther. 2019 Dec 11. doi: 10.1038/s41434-019-0113-4. [Epub ahead of print]

PMID:
31827259
3.

Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases.

Poletto E, Pasqualim G, Giugliani R, Matte U, Baldo G.

Genet Mol Biol. 2019;42(1 suppl 1):261-285. doi: 10.1590/1678-4685-GMB-2018-0100. Epub 2019 May 23.

5.

Nasal Administration of Cationic Nanoemulsions as Nucleic Acids Delivery Systems Aiming at Mucopolysaccharidosis Type I Gene Therapy.

Schuh RS, Bidone J, Poletto E, Pinheiro CV, Pasqualim G, de Carvalho TG, Farinon M, da Silva Diel D, Xavier RM, Baldo G, Matte U, Teixeira HF.

Pharm Res. 2018 Sep 26;35(11):221. doi: 10.1007/s11095-018-2503-5.

PMID:
30259180
6.

In vivo genome editing of mucopolysaccharidosis I mice using the CRISPR/Cas9 system.

Schuh RS, Poletto É, Pasqualim G, Tavares AMV, Meyer FS, Gonzalez EA, Giugliani R, Matte U, Teixeira HF, Baldo G.

J Control Release. 2018 Oct 28;288:23-33. doi: 10.1016/j.jconrel.2018.08.031. Epub 2018 Aug 28.

PMID:
30170069
7.

Physicochemical properties of cationic nanoemulsions and liposomes obtained by microfluidization complexed with a single plasmid or along with an oligonucleotide: Implications for CRISPR/Cas technology.

Schuh RS, Poletto É, Fachel FNS, Matte U, Baldo G, Teixeira HF.

J Colloid Interface Sci. 2018 Nov 15;530:243-255. doi: 10.1016/j.jcis.2018.06.058. Epub 2018 Jun 22.

PMID:
29982016
8.

Intra-articular nonviral gene therapy in mucopolysaccharidosis I mice.

Bidone J, Schuh RS, Farinon M, Poletto É, Pasqualim G, de Oliveira PG, Fraga M, Xavier RM, Baldo G, Teixeira HF, Matte U.

Int J Pharm. 2018 Sep 5;548(1):151-158. doi: 10.1016/j.ijpharm.2018.06.049. Epub 2018 Jun 27.

PMID:
29940298
9.

Cathepsin B inhibition attenuates cardiovascular pathology in mucopolysaccharidosis I mice.

Gonzalez EA, Martins GR, Tavares AMV, Viegas M, Poletto E, Giugliani R, Matte U, Baldo G.

Life Sci. 2018 Mar 1;196:102-109. doi: 10.1016/j.lfs.2018.01.020. Epub 2018 Jan 31.

PMID:
29366749
10.

Losartan improves aortic dilatation and cardiovascular disease in mucopolysaccharidosis I.

Gonzalez EA, Tavares AM, Poletto E, Giugliani R, Matte U, Baldo G.

J Inherit Metab Dis. 2017 May;40(3):311-312. doi: 10.1007/s10545-017-0014-x. Epub 2017 Feb 3. No abstract available.

PMID:
28160112
11.

Progressive heart disease in mucopolysaccharidosis type I mice may be mediated by increased cathepsin B activity.

Baldo G, Tavares AM, Gonzalez E, Poletto E, Mayer FQ, Matte UD, Giugliani R.

Cardiovasc Pathol. 2017 Mar - Apr;27:45-50. doi: 10.1016/j.carpath.2017.01.001. Epub 2017 Jan 6.

PMID:
28104572

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