2013

Zenack D, Schouwey K, Chen D, Ekström P, Tanger E, Bremner R, van Lohuizen M, Arsenijevic Y (2012). Retinal degeneration depends on Bmi1 function and reactivation of cell cycle proteins. Proc Natl Acad Sci U S A.  [Epub ahead of print]  >>

2011

Allocca M, Manfredi A, Iodice C, Di Vicino U, Auricchio A (2011). AAV-mediated gene replacement either alone or in combination with physical and pharmacological agents results in partial and transient protection from photoreceptor degeneration associated with βPDE deficiency. Invest Ophthalmol Vis Sci 52: 5713-5719. >>

Carvalho LS, Xu J, Pearson RA, Smith AJ, Bainbridge JW, Morris LM, Fliesler SJ, Ding XQ, Ali RR (2011). Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy. Hum Mol Genet 20: 3161-75. >>

Colella P, Iodice C, Di Vicino U, Annunziata I, Surace EM, Auricchio A (2011). Non-erythropoietic erytropoietin derivates protect from light-induced and genetic photoreceptor degeneration. Hum Mol Genet 20: 2251-2262. >>

Karali M, Manfredi A, Puppo A, Marrocco E, Gargiulo A, Allocca MC, Della Corte M, Rossi S, Giunti M, Bacci ML, Simonelli F, Surace EM, Banfi S, Auricchio A (2011). microRNA-restricted transgene expression in the retina. PLoS ONE 6: e22166. >>

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 (2011). AAV-mediated photoreceptor transduction of the pig cone-enriched retina. Gene Ther 18: 637-645. >>

Mussolino C, Sanges D, Marrocco E, Bonetti C, Di Vicino U, Marigo V, Auricchio A, Meroni G, Surace EM (2011). Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa. EMBO Mol Med 3: 1-11. >>

Testa F, Surace EM, Rossi S, Marrocco E, Gargiulo A, Di Iorio V, Ziviello C, Nesti A, Fecarotta S, Bacci ML, Giunti M, Corte MD, Banfi S, Auricchio A, Simonelli F (2011). Evaluation of Italian Patients with Leber Congenital Amaurosis due to AIPL1 Mutations Highlights the Potential Applicability of Gene Therapy. Invest Ophthalmol Vis Sci 52: 5618-5624. >>

2010

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 G-S, Ziviello C, Acerra C, Wright JF, Wellman McDonnell J, High KA, Bennett J, Auricchio A (2010). Gene Therapy for Leber’s Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration. Mol Ther 18: 643-650. >>

Sun X, Pawlyk B, Xu X, Liu X, Bulgakov OV, Adamian M, Sandberg MA, Khani SC, Tan MH, Smith AJ, Ali RR, Li T (2010). Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations. Gene Ther 17: 117-131. >>

2009

Maguire AM, High, KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying G-S, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JIW, Hauck B, Zelanaia O, Zhu X, Raffini L, Coppieters F, De Baere E, Shindler KS, Volpe NJ, Surace EM, Acerra C, Lyubarsky A, Redmond TM, Stone E, Sun J, Wellman McDonnell J, Leroy BP, Simonelli F, Bennett J (2009). Age-dependent effects of RPE65 gene therapy for Leber’s congenital amaurosis: a phase 1 dose escalation trial. Lancet 374: 1597-1605. >>

Tan MH, Smith AJ, Pawlyk B, Xu X, Liu X, Bainbridge JB, Basche M, McIntosh J, Tran HV, Nathwani A, Li T, Ali RR (2009). Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors. Hum Mol Genet 18: 2099-2114. >>