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154 results

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Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease.
Trapani I, Toriello E, de Simone S, Colella P, Iodice C, Polishchuk EV, Sommella A, Colecchi L, Rossi S, Simonelli F, Giunti M, Bacci ML, Polishchuk RS, Auricchio A. Trapani I, et al. Among authors: bacci ml. Hum Mol Genet. 2015 Dec 1;24(23):6811-25. doi: 10.1093/hmg/ddv386. Epub 2015 Sep 29. Hum Mol Genet. 2015. PMID: 26420842 Free PMC article.
Combined rod and cone transduction by adeno-associated virus 2/8.
Manfredi A, Marrocco E, Puppo A, Cesi G, Sommella A, Della Corte M, Rossi S, Giunti M, Craft CM, Bacci ML, Simonelli F, Surace EM, Auricchio A. Manfredi A, et al. Among authors: bacci ml. Hum Gene Ther. 2013 Dec;24(12):982-92. doi: 10.1089/hum.2013.154. Epub 2013 Oct 30. Hum Gene Ther. 2013. PMID: 24067103 Free PMC article.
A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-associated Virus Serotypes Upon Cerebrospinal Fluid Administration in Pigs.
Sorrentino NC, Maffia V, Strollo S, Cacace V, Romagnoli N, Manfredi A, Ventrella D, Dondi F, Barone F, Giunti M, Graham AR, Huang Y, Kalled SL, Auricchio A, Bacci ML, Surace EM, Fraldi A. Sorrentino NC, et al. Among authors: bacci ml. Mol Ther. 2016 Feb;24(2):276-286. doi: 10.1038/mt.2015.212. Epub 2015 Dec 7. Mol Ther. 2016. PMID: 26639405 Free PMC article.
MicroRNA-restricted transgene expression in the retina.
Karali M, Manfredi A, Puppo A, Marrocco E, Gargiulo A, Allocca M, Corte MD, Rossi S, Giunti M, Bacci ML, Simonelli F, Surace EM, Banfi S, Auricchio A. Karali M, et al. Among authors: bacci ml. PLoS One. 2011;6(7):e22166. doi: 10.1371/journal.pone.0022166. Epub 2011 Jul 26. PLoS One. 2011. PMID: 21818300 Free PMC article.
AAV-mediated photoreceptor transduction of the pig cone-enriched retina.
Mussolino C, della Corte M, Rossi S, Viola F, Di Vicino U, Marrocco E, Neglia S, Doria M, Testa F, Giovannoni R, Crasta M, Giunti M, Villani E, Lavitrano M, Bacci ML, Ratiglia R, Simonelli F, Auricchio A, Surace EM. Mussolino C, et al. Among authors: bacci ml. Gene Ther. 2011 Jul;18(7):637-45. doi: 10.1038/gt.2011.3. Epub 2011 Mar 17. Gene Ther. 2011. PMID: 21412286 Free PMC article.
Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors.
Colella P, Trapani I, Cesi G, Sommella A, Manfredi A, Puppo A, Iodice C, Rossi S, Simonelli F, Giunti M, Bacci ML, Auricchio A. Colella P, et al. Among authors: bacci ml. Gene Ther. 2014 Apr;21(4):450-6. doi: 10.1038/gt.2014.8. Epub 2014 Feb 27. Gene Ther. 2014. PMID: 24572793
Rhodopsin targeted transcriptional silencing by DNA-binding.
Botta S, Marrocco E, de Prisco N, Curion F, Renda M, Sofia M, Lupo M, Carissimo A, Bacci ML, Gesualdo C, Rossi S, Simonelli F, Surace EM. Botta S, et al. Among authors: bacci ml. Elife. 2016 Mar 14;5:e12242. doi: 10.7554/eLife.12242. Elife. 2016. PMID: 26974343 Free PMC article.
Evaluation of Italian patients with leber congenital amaurosis due to AIPL1 mutations highlights the potential applicability of gene therapy.
Testa F, Surace EM, Rossi S, Marrocco E, Gargiulo A, Di Iorio V, Ziviello C, Nesti A, Fecarotta S, Bacci ML, Giunti M, Della Corte M, Banfi S, Auricchio A, Simonelli F. Testa F, et al. Among authors: bacci ml. Invest Ophthalmol Vis Sci. 2011 Jul 29;52(8):5618-24. doi: 10.1167/iovs.10-6543. Invest Ophthalmol Vis Sci. 2011. PMID: 21474771
154 results