Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement
Cideciyan, Artur V., Jacobson, Samuel G., Beltran, William A., Sumaroka, Alexander, Swider, Malgorzata, Iwabe, Simone, Roman, Alejandro J., Olivares, Melani B., Schwartz, Sharon B., Komáromy, András M., Hauswirth, William W., Aguirre, Gustavo D.
Published in Proceedings of the National Academy of Sciences - PNAS (05.02.2013)
Published in Proceedings of the National Academy of Sciences - PNAS (05.02.2013)
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Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy
Boye, Shannon E, Huang, Wei-Chieh, Roman, Alejandro J, Sumaroka, Alexander, Boye, Sanford L, Ryals, Renee C, Olivares, Melani B, Ruan, Qing, Tucker, Budd A, Stone, Edwin M, Swaroop, Anand, Cideciyan, Artur V, Hauswirth, William W, Jacobson, Samuel G
Published in PloS one (26.03.2014)
Published in PloS one (26.03.2014)
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Determining consequences of retinal membrane guanylyl cyclase (RetGC1) deficiency in human Leber congenital amaurosis en route to therapy: residual cone-photoreceptor vision correlates with biochemical properties of the mutants
Jacobson, Samuel G, Cideciyan, Artur V, Peshenko, Igor V, Sumaroka, Alexander, Olshevskaya, Elena V, Cao, Lihui, Schwartz, Sharon B, Roman, Alejandro J, Olivares, Melani B, Sadigh, Sam, Yau, King-Wai, Heon, Elise, Stone, Edwin M, Dizhoor, Alexander M
Published in Human molecular genetics (01.01.2013)
Published in Human molecular genetics (01.01.2013)
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Human CRB1-associated retinal degeneration: comparison with the rd8 Crb1-mutant mouse model
Aleman, Tomas S, Cideciyan, Artur V, Aguirre, Geoffrey K, Huang, Wei Chieh, Mullins, Cristina L, Roman, Alejandro J, Sumaroka, Alexander, Olivares, Melani B, Tsai, Frank F, Schwartz, Sharon B, Vandenberghe, Luk H, Limberis, Maria P, Stone, Edwin M, Bell, Peter, Wilson, James M, Jacobson, Samuel G
Published in Investigative ophthalmology & visual science (29.08.2011)
Published in Investigative ophthalmology & visual science (29.08.2011)
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Retinal disease course in Usher syndrome 1B due to MYO7A mutations
Jacobson, Samuel G, Cideciyan, Artur V, Gibbs, Dan, Sumaroka, Alexander, Roman, Alejandro J, Aleman, Tomas S, Schwartz, Sharon B, Olivares, Melani B, Russell, Robert C, Steinberg, Janet D, Kenna, Margaret A, Kimberling, William J, Rehm, Heidi L, Williams, David S
Published in Investigative ophthalmology & visual science (07.10.2011)
Published in Investigative ophthalmology & visual science (07.10.2011)
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Intervisit variability of visual parameters in Leber congenital amaurosis caused by RPE65 mutations
Roman, Alejandro J, Cideciyan, Artur V, Schwartz, Sharon B, Olivares, Melani B, Heon, Elise, Jacobson, Samuel G
Published in Investigative ophthalmology & visual science (15.02.2013)
Published in Investigative ophthalmology & visual science (15.02.2013)
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Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years
Jacobson, Samuel G, Cideciyan, Artur V, Ratnakaram, Ramakrishna, Heon, Elise, Schwartz, Sharon B, Roman, Alejandro J, Peden, Marc C, Aleman, Tomas S, Boye, Sanford L, Sumaroka, Alexander, Conlon, Thomas J, Calcedo, Roberto, Pang, Ji-Jing, Erger, Kirsten E, Olivares, Melani B, Mullins, Cristina L, Swider, Malgorzata, Kaushal, Shalesh, Feuer, William J, Iannaccone, Alessandro, Fishman, Gerald A, Stone, Edwin M, Byrne, Barry J, Hauswirth, William W
Published in Archives of ophthalmology (1960) (01.01.2012)
Published in Archives of ophthalmology (1960) (01.01.2012)
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Natural History of Cone Disease in the Murine Model of Leber Congenital Amaurosis Due to CEP290 Mutation: Determining the Timing and Expectation of Therapy: e92928
Boye, Shannon E, Huang, Wei-Chieh, Roman, Alejandro J, Sumaroka, Alexander, Boye, Sanford L, Ryals, Renee C, Olivares, Melani B, Ruan, Qing, Tucker, Budd A, Stone, Edwin M
Published in PloS one (01.03.2014)
Published in PloS one (01.03.2014)
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