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miR-181a/b downregulation: a mutation-independent therapeutic approach for inherited retinal diseases.
Carrella S, Di Guida M, Brillante S, Piccolo D, Ciampi L, Guadagnino I, Garcia Piqueras J, Pizzo M, Marrocco E, Molinari M, Petrogiannakis G, Barbato S, Ezhova Y, Auricchio A, Franco B, De Leonibus E, Surace EM, Indrieri A, Banfi S. Carrella S, et al. Among authors: banfi s. EMBO Mol Med. 2022 Nov 8;14(11):e15941. doi: 10.15252/emmm.202215941. Epub 2022 Oct 4. EMBO Mol Med. 2022. PMID: 36194668 Free PMC article.
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.
Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, Rossi S, Marshall K, Banfi S, Surace EM, Sun J, Redmond TM, Zhu X, Shindler KS, Ying GS, Ziviello C, Acerra C, Wright JF, McDonnell JW, High KA, Bennett J, Auricchio A. Simonelli F, et al. Among authors: banfi s. Mol Ther. 2010 Mar;18(3):643-50. doi: 10.1038/mt.2009.277. Epub 2009 Dec 1. Mol Ther. 2010. PMID: 19953081 Free PMC article. Clinical Trial.
miR-204 is required for lens and retinal development via Meis2 targeting.
Conte I, Carrella S, Avellino R, Karali M, Marco-Ferreres R, Bovolenta P, Banfi S. Conte I, et al. Among authors: banfi s. Proc Natl Acad Sci U S A. 2010 Aug 31;107(35):15491-6. doi: 10.1073/pnas.0914785107. Epub 2010 Aug 16. Proc Natl Acad Sci U S A. 2010. PMID: 20713703 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: banfi s. Invest Ophthalmol Vis Sci. 2011 Jul 29;52(8):5618-24. doi: 10.1167/iovs.10-6543. Invest Ophthalmol Vis Sci. 2011. PMID: 21474771
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: banfi s. PLoS One. 2011;6(7):e22166. doi: 10.1371/journal.pone.0022166. Epub 2011 Jul 26. PLoS One. 2011. PMID: 21818300 Free PMC article.
265 results