The dose-response relationship of subretinal gene therapy with rAAV2tYF-CB-hRS1 in a mouse model of X-linked retinoschisis by Salma Hassan, Salma Hassan, Ying Hsu, Jacob M. Thompson, Jacob M. Thompson, Emily Kalmanek, Joel A. VandeLune, Sarah Stanley, Arlene V. Drack, Arlene V. Drack, Arlene V. Drack

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TRIP8b is required for maximal expression of HCN1 in the mouse retina. by Yuan Pan, Sajag Bhattarai, Modestos Modestou, Arlene V Drack, Dane M Chetkovich, Sheila A Baker

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Alström syndrome caused by maternal uniparental disomy by Madeline Q.R. Lopour, Lisa A. Schimmenti, Nicole J. Boczek, Hutton M. Kearney, Arlene V. Drack, Michael C. Brodsky

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Ectopic expression of human BBS4 can rescue Bardet-Biedl syndrome phenotypes in Bbs4 null mice. by Xitiz Chamling, Seongjin Seo, Kevin Bugge, Charles Searby, Deng F Guo, Arlene V Drack, Kamal Rahmouni, Val C Sheffield

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AAV2/4-RS1 gene therapy in the retinoschisin knockout mouse model of X-linked retinoschisis. by Brittni A Scruggs, Sajag Bhattarai, Megan Helms, Ioana Cherascu, Adisa Salesevic, Elliot Stalter, Joseph Laird, Sheila A Baker, Arlene V Drack

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AAV2/4-RS1 gene therapy in the retinoschisin knockout mouse model of X-linked retinoschisis by Brittni A. Scruggs, Sajag Bhattarai, Megan Helms, Ioana Cherascu, Adisa Salesevic, Elliot Stalter, Joseph Laird, Sheila A. Baker, Arlene V. Drack

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Nanophthalmos patient with a THR518MET mutation in MYRF, a case report by Joshua Hagedorn, Armin Avdic, Michael J. Schnieders, Benjamin R. Roos, Young H. Kwon, Arlene V. Drack, Erin A. Boese, John H. Fingert

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Patient-specific iPSC-derived photoreceptor precursor cells as a means to investigate retinitis pigmentosa by Budd A Tucker, Robert F Mullins, Luan M Streb, Kristin Anfinson, Mari E Eyestone, Emily Kaalberg, Megan J Riker, Arlene V Drack, Terry A Braun, Edwin M Stone

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A novel porcine model of CLN3 Batten disease recapitulates clinical phenotypes by Vicki J. Swier, Katherine A. White, Tyler B. Johnson, Xiaojun Wang, Jimin Han, David A. Pearce, Ruchira Singh, Arlene V. Drack, Wanda Pfeifer, Christopher S. Rogers, Jon J. Brudvig, Jill M. Weimer

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Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model by Ying Hsu, Sajag Bhattarai, Jacob M. Thompson, Angela Mahoney, Jacintha Thomas, Sara K. Mayer, Poppy Datta, Janelle Garrison, Charles C. Searby, Luk H. Vandenberghe, Seongjin Seo, Val C. Sheffield, Arlene V. Drack

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Progressive retinal degeneration of rods and cones in a Bardet-Biedl syndrome type 10 mouse model by Sara K. Mayer, Jacintha Thomas, Megan Helms, Aishwarya Kothapalli, Ioana Cherascu, Adisa Salesevic, Elliot Stalter, Kai Wang, Poppy Datta, Charles Searby, Seongjin Seo, Ying Hsu, Sajag Bhattarai, Val C. Sheffield, Arlene V. Drack

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Long-term functional and structural outcomes in X-linked retinoschisis: implications for clinical trials by Beau J. Fenner, Beau J. Fenner, Beau J. Fenner, Beau J. Fenner, Beau J. Fenner, Jonathan F. Russell, Jonathan F. Russell, Arlene V. Drack, Arlene V. Drack, Alina V. Dumitrescu, Alina V. Dumitrescu, Elliott H. Sohn, Elliott H. Sohn, Stephen R. Russell, Stephen R. Russell, H. Culver Boldt, H. Culver Boldt, Louisa M. Affatigato, Jeremy M. Hoffmann, Jeaneen L. Andorf, Edwin M. Stone, Edwin M. Stone, Ian C. Han, Ian C. Han

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Endothelial BBSome is essential for vascular, metabolic, and retinal functions by Jingwei Jiang, John J. Reho, Sajag Bhattarai, Ioana Cherascu, Adam Hedberg-Buenz, Kacie J. Meyer, Fariba Tayyari, Adam J. Rauckhorst, Deng Fu Guo, Donald A. Morgan, Eric B. Taylor, Michael G. Anderson, Arlene V. Drack, Kamal Rahmouni

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Restoration of Cone Sensitivity to Individuals with Congenital Photoreceptor Blindness within the Phase 1/2 Sepofarsen Trial by Artur V. Cideciyan, PhD, Samuel G. Jacobson, MD, PhD, Allen C. Ho, MD, Arun K. Krishnan, OD, PhD, Alejandro J. Roman, MS, Alexandra V. Garafalo, MS, Vivian Wu, MS, Malgorzata Swider, PhD, Alexander Sumaroka, PhD, Caroline Van Cauwenbergh, PhD, Stephen R. Russell, MD, Arlene V. Drack, MD, Bart P. Leroy, MD, PhD, Michael R. Schwartz, MS, Aniz Girach, MD

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