Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement von 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.

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Identification of circular RNAs hosted by the RPGR ORF15 genomic locus von Appelbaum, Tatyana, Aguirre, Gustavo D., Beltran, William A.

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Canine retina has a primate fovea-like bouquet of cone photoreceptors which is affected by inherited macular degenerations von Beltran, William A, Cideciyan, Artur V, Guziewicz, Karina E, Iwabe, Simone, Swider, Malgorzata, Scott, Erin M, Savina, Svetlana V, Ruthel, Gordon, Stefano, Frank, Zhang, Lingli, Zorger, Richard, Sumaroka, Alexander, Jacobson, Samuel G, Aguirre, Gustavo D

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Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa von Beltran, William A., Cideciyan, Artur V., Lewin, Alfred S., Iwabe, Simone, Khanna, Hemant, Sumaroka, Alexander, Chiodo, Vince A., Fajardo, Diego S., Román, Alejandro J., Deng, Wen-Tao, Swider, Malgorzata, Alemán, Tomas S., Boye, Sanford L., Genini, Sem, Swaroop, Anand, Hauswirth, William W., Jacobson, Samuel G., Aguirre, Gustavo D.

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Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis von Aguirre, Gustavo D., Cideciyan, Artur V., Dufour, Valérie L., Ripolles-García, Ana, Sudharsan, Raghavi, Swider, Malgorzata, Nikonov, Roman, Iwabe, Simone, Boye, Sanford L., Hauswirth, William W., Jacobson, Samuel G., Beltran, William A.

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Up-regulation of tumor necrosis factor superfamily genes in early phases of photoreceptor degeneration von Genini, Sem, Beltran, William A, Aguirre, Gustavo D

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Restoration of visual function by expression of a light-gated mammalian ion channel in retinal ganglion cells or ON-bipolar cells von Gaub, Benjamin M., Berry, Michael H., Holt, Amy E., Reiner, Andreas, Kienzler, Michael A., Dolgova, Natalia, Nikonov, Sergei, Aguirre, Gustavo D., Beltran, William A., Flannery, John G., Isacoff, Ehud Y.

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Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease von Beltran, William A., Cideciyan, Artur V., Iwabe, Simone, Swider, Malgorzata, Kosyk, Mychajlo S., McDaid, Kendra, Martynyuk, Inna, Ying, Gui-Shuang, Shaffer, James, Deng, Wen-Tao, Boye, Sanford L., Lewin, Alfred S., Hauswirth, William W., Jacobson, Samuel G., Aguirre, Gustavo D.

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Retinal prolactin isoform PRLΔE1 sustains rod disease in inherited retinal degenerations von Sudharsan, Raghavi, Kwok, Jennifer, Swider, Malgorzata, Sumaroka, Alexander, Aguirre, Gustavo D., Cideciyan, Artur V., Beltran, William A.

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Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects von Guziewicz, Karina E, Zangerl, Barbara, Komáromy, András M, Iwabe, Simone, Chiodo, Vincent A, Boye, Sanford L, Hauswirth, William W, Beltran, William A, Aguirre, Gustavo D

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scAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution von Öztürk, Bilge E, Johnson, Molly E, Kleyman, Michael, Turunç, Serhan, He, Jing, Jabalameli, Sara, Xi, Zhouhuan, Visel, Meike, Dufour, Valérie L, Iwabe, Simone, Pompeo Marinho, Luis Felipe L, Aguirre, Gustavo D, Sahel, José-Alain, Schaffer, David V, Pfenning, Andreas R, Flannery, John G, Beltran, William A, Stauffer, William R, Byrne, Leah C

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Photoreceptor function and structure in retinal degenerations caused by biallelic BEST1 mutations von Cideciyan, Artur V., Jacobson, Samuel G., Sumaroka, Alexander, Swider, Malgorzata, Krishnan, Arun K., Sheplock, Rebecca, Garafalo, Alexandra V., Guziewicz, Karina E., Aguirre, Gustavo D., Beltran, William A., Matsui, Yoshitsugu, Kondo, Mineo, Heon, Elise

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Exclusion of the unfolded protein response in light-induced retinal degeneration in the canine T4R RHO model of autosomal dominant retinitis pigmentosa von Marsili, Stefania, Genini, Sem, Sudharsan, Raghavi, Gingrich, Jeremy, Aguirre, Gustavo D, Beltran, William A

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Enhancer of Zeste Homolog 2 (EZH2) Contributes to Rod Photoreceptor Death Process in Several Forms of Retinal Degeneration and Its Activity Can Serve as a Biomarker for Therapy Eff... von Mbefo, Martial, Berger, Adeline, Schouwey, Karine, Gerard, Xavier, Kostic, Corinne, Beryozkin, Avigail, Sharon, Dror, Dolfuss, Helene, Munier, Francis, Hoai Viet Tran, van Lohuizen, Maarten, Beltran, William A., Arsenijevic, Yvan

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Short prolactin isoforms are expressed in photoreceptors of canine retinas undergoing retinal degeneration von Sudharsan, Raghavi, Murgiano, Leonardo, Tang, Hsin-Yao, Olsen, Timothy W., Chavali, Venkata R. M., Aguirre, Gustavo D., Beltran, William A.

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Transient Photoreceptor Deconstruction by CNTF Enhances rAAV-Mediated Cone Functional Rescue in Late Stage CNGB3-Achromatopsia von Komáromy, András M, Rowlan, Jessica S, Corr, Amanda T Parton, Reinstein, Shelby L, Boye, Sanford L, Cooper, Ann E, Gonzalez, Amaliris, Levy, Britt, Wen, Rong, Hauswirth, William W, Beltran, William A, Aguirre, Gustavo D

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Photochemical Restoration of Light Sensitivity in the Degenerated Canine Retina von Nikonov, Sergei, Dolgova, Natalia, Sudharsan, Raghavi, Tochitsky, Ivan, Iwabe, Simone, Guzman, Jose-Manuel, Van Gelder, Russell N, Kramer, Richard H, Aguirre, Gustavo D, Beltran, William A

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An optimized workflow for transcriptomic analysis from archival paraformaldehyde-fixed retinal tissues collected by laser capture microdissection von Takahashi, Kei, Beltran, William A., Sudharsan, Raghavi

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Characterization of the Canine Retinal Vasculature With Optical Coherence Tomography Angiography: Comparisons With Histology and Fluorescein Angiography von Ripolles-Garcia, Ana, Ruthel, Gordon, Ying, Gui-Shuang, Chen, Yineng, Cuenca, Nicolas, Aguirre, Gustavo D., Beltran, William A.

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Human cone photoreceptor dependence on RPE65 isomerase von Jacobson, Samuel G, Aleman, Tomas S, Cideciyan, Artur V, Heon, Elise, Golczak, Marcin, Beltran, William A, Sumaroka, Alexander, Schwartz, Sharon B, Roman, Alejandro J, Windsor, Elizabeth A.M, Wilson, James M, Aguirre, Gustavo D, Stone, Edwin M, Palczewski, Krzysztof

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