Progress in treating inherited retinal diseases: Early subretinal gene therapy clinical trials and candidates for future initiatives von Garafalo, Alexandra V., Cideciyan, Artur V., Héon, Elise, Sheplock, Rebecca, Pearson, Alexander, WeiYang Yu, Caberry, Sumaroka, Alexander, Aguirre, Gustavo D., Jacobson, Samuel G.

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Strong upregulation of inflammatory genes accompanies photoreceptor demise in canine models of retinal degeneration von Appelbaum, Tatyana, Santana, Evelyn, 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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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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Natural disease history of a canine model of oligogenic RPGRIP1-cone-rod dystrophy establishes variable effects of previously and newly mapped modifier loci von Ripolles-Garcia, Ana, Murgiano, Leonardo, Ziolkowska, Natalia, Marinho, Felipe Pompeo, Roszak, Karolina, Iffrig, Sommer, Aguirre, Gustavo D, Miyadera, Keiko

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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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Formal commentary von Aguirre, Gustavo D, Lohi, Hannes, Kaukonen, Maria, Murgiano, Leonardo

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Bestrophinopathy: An RPE-photoreceptor interface disease von Guziewicz, Karina E., Sinha, Divya, Gómez, Néstor M., Zorych, Kathryn, Dutrow, Emily V., Dhingra, Anuradha, Mullins, Robert F., Stone, Edwin M., Gamm, David M., Boesze-Battaglia, Kathleen, Aguirre, Gustavo D.

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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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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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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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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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A digital atlas of the dog brain von Datta, Ritobrato, Lee, Jongho, Duda, Jeffrey, Avants, Brian B, Vite, Charles H, Tseng, Ben, Gee, James C, Aguirre, Gustavo D, Aguirre, Geoffrey K

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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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Molecular characterization of MAP9 in the photoreceptor sensory cilia as a modifier in canine RPGRIP1 -associated cone-rod dystrophy von Takahashi, Kei, Kwok, Jennifer C, Sato, Yu, Aguirre, Gustavo D, Miyadera, Keiko

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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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Genetic and phenotypic variations of inherited retinal diseases in dogs: the power of within- and across-breed studies von Miyadera, Keiko, Acland, Gregory M, Aguirre, Gustavo D

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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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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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A Naturally Occurring Canine Model of Autosomal Recessive Congenital Stationary Night Blindness von Kondo, Mineo, Das, Gautami, Imai, Ryoetsu, Santana, Evelyn, Nakashita, Tomio, Imawaka, Miho, Ueda, Kosuke, Ohtsuka, Hirohiko, Sakai, Kazuhiko, Aihara, Takehiro, Kato, Kumiko, Sugimoto, Masahiko, Ueno, Shinji, Nishizawa, Yuji, Aguirre, Gustavo D, Miyadera, Keiko

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