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Human cone photoreceptor transplantation stimulates remodeling and restores function in AIPL1 model of end-stage Leber congenital amaurosis Procyk, Christopher A.; Melati, Anna; Ribeiro, Joana; Liu, Jingshu; Branch, Matthew J.; Delicata, Jamie D.; Tariq, Menahil; Kalarygrou, Aikaterini A.; Kapadia, Jessica; Khorsani, Majid Moshtagh; West, Emma L.; Smith, Alexander J.; Gonzalez-Cordero, Anai; Ali, Robin R.; Pearson, Rachael A. Share Save
Phase 1/2 AAV5-hRKp.RPGR RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa Michaelides, Michel; Besirli, Cagri G.; Yang, Yesa; De Guimaraes, Thales A. C.; Wong, Sui Chien; Huckfeldt, Rachel M.; Comander, Jason I.; Sahel, Jose-Alain; Shah, Syed Mahmood; Tee, James J. L.; Kumaran, Neruban; Georgiadis, Anastasios; Minnick, Pansy; Zeldin, Robert; Naylor, Stuart; Xu, Jialin; Clark, Michael; Anglade, Eddy; Wong, Peggy; Fleck, Penny R.; Fung, Albert; Peluso, Colleen; Kalitzeos, Angelos; Georgiou, Michalis; Ripamonti, Caterina; Smith, Alexander J.; Ali, Robin R.; Forbes, Alexandria; Bainbridge, James Share Save
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Restoration of vision after transplantation of photoreceptors (vol 485, pg 99, 2012) Pearson, R. A.; Barber, A. C.; Rizzi, M.; Hippert, C.; Xue, T.; West, E. L.; Duran, Y.; Smith, A. J.; Chuang, J. Z.; Azam, S. A.; Luhmann, U. F. O.; Benucci, A.; Sung, C. H.; Bainbridge, J. W.; Carandini, M.; Yau, K. -W.; Sowden, J. C.; Ali, R. R. Share Save
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PRPF8-mediated dysregulation of hBrr2 helicase disrupts human spliceosome kinetics and 5-splice-site selection causing tissue-specific defects Atkinson, Robert; Georgiou, Maria; Yang, Chunbo; Szymanska, Katarzyna; Lahat, Albert; Vasconcelos, Elton J. R.; Ji, Yanlong; Molina, Marina Moya; Collin, Joseph; Queen, Rachel; Dorgau, Birthe; Watson, Avril; Kurzawa-Akanbi, Marzena; Laws, Ross; Saxena, Abhijit; Beh, Chia Shyan; Siachisumo, Chileleko; Goertler, Franziska; Karwatka, Magdalena; Davey, Tracey; Inglehearn, Chris F.; Mckibbin, Martin; Luehrmann, Reinhard; Steel, David H.; Elliott, David J.; Armstrong, Lyle; Urlaub, Henning; Ali, Robin R.; Grellscheid, Sushma-Nagaraja; Johnson, Colin A.; Mozaffari-Jovin, Sina; Lako, Majlinda Share Save
First-in-Human Gene Therapy Trial of AAV8-hCARp.h CNGB3 in Adults and Children With CNGB3-associated Achromatopsia Michaelides, Michel; Hirji, Nashila; Wong, Sui Chien; Besirli, Cagri G.; Zaman, Serena; Kumaran, Neruban; Georgiadis, Anastasios; Smith, Alexander J.; Ripamonti, Caterina; Gottlob, Irene; Robson, Anthony G.; Thiadens, Alberta; Henderson, Robert H.; Fleck, Penny; Anglade, Eddy; Dong, Xiangwen; Capuano, George; Lu, Wentao; Berry, Pamela; Kane, Thomas; Naylor, Stuart; Georgiou, Michalis; Kalitzeos, Angelos; Ali, Robin R.; Forbes, Alexandria; Bainbridge, James Share Save
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Spatiotemporal control of actomyosin contractility by MRCKβ signaling drives phagocytosis Zihni, Ceniz; Georgiadis, Anastasios; Ramsden, Conor M.; Sanchez-Heras, Elena; Haas, Alexis J.; Nommiste, Britta; Semenyuk, Olha; Bainbridge, James W. B.; Coffey, Peter J.; Smith, Alexander J.; Ali, Robin R.; Balda, Maria S.; Matter, Karl Share Save
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Differentiation of brain and retinal organoids from confluent cultures of pluripotent stem cells connected by nerve-like axonal projections of optic origin Fernando, Milan; Lee, Scott; Wark, Jesse R.; Xiao, Di; Lim, Benjamin Y.; O'Hara-Wright, Michelle; Kim, Hani J.; Smith, Grady C.; Wong, Ted; Teber, Erdahl T.; Ali, Robin R.; Yang, Pengyi; Graham, Mark E.; Gonzalez-Cordero, Anai Share Save