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Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases.
Acta Paediatr. 2012 Jul;101(7):692-701. doi: 10.1111/j.1651-2227.2012.02674.x. Epub 2012 Apr 11.
Acta Paediatr. 2012.
PMID: 22428546
Review.
In vivo delivery of naked antisense oligos in aged mdx mice: analysis of dystrophin restoration in skeletal and cardiac muscle.
Vitiello L, Bassi N, Campagnolo P, Zaccariotto E, Occhi G, Malerba A, Pigozzo S, Reggiani C, Ausoni S, Zaglia T, Gamba P, Baroni MD, Ditadi AP.
Vitiello L, et al. Among authors: zaccariotto e.
Neuromuscul Disord. 2008 Aug;18(8):597-605. doi: 10.1016/j.nmd.2008.05.011. Epub 2008 Jul 3.
Neuromuscul Disord. 2008.
PMID: 18602263
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A novel brain-penetrant oral UGT8 inhibitor decreases in vivo galactosphingolipid biosynthesis in murine Krabbe disease.
Zaccariotto E, Cachón-González MB, Wang B, Lim S, Hirth B, Park H, Fezoui M, Sardi SP, Mason P, Barker RH Jr, Cox TM.
Zaccariotto E, et al.
Biomed Pharmacother. 2022 May;149:112808. doi: 10.1016/j.biopha.2022.112808. Epub 2022 Mar 12.
Biomed Pharmacother. 2022.
PMID: 35290889
Free article.
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Genetics and Therapies for GM2 Gangliosidosis.
Cachon-Gonzalez MB, Zaccariotto E, Cox TM.
Cachon-Gonzalez MB, et al. Among authors: zaccariotto e.
Curr Gene Ther. 2018;18(2):68-89. doi: 10.2174/1566523218666180404162622.
Curr Gene Ther. 2018.
PMID: 29618308
Free PMC article.
Review.
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