Losing Ceci von Sanford, Ethan L

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Continuously Cumulating Meta-Analysis and Replicability von Braver, Sanford L., Thoemmes, Felix J., Rosenthal, Robert

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RNA Splicing and Disease: Animal Models to Therapies von Montes, Matías, Sanford, Brianne L., Comiskey, Daniel F., Chandler, Dawn S.

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Current Clinical Applications of In Vivo Gene Therapy with AAVs von Mendell, Jerry R., Al-Zaidy, Samiah A., Rodino-Klapac, Louise R., Goodspeed, Kimberly, Gray, Steven J., Kay, Christine N., Boye, Sanford L., Boye, Shannon E., George, Lindsey A., Salabarria, Stephanie, Corti, Manuela, Byrne, Barry J., Tremblay, Jacques P.

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Mechanisms of telomerase inhibition by oxidized and therapeutic dNTPs von Sanford, Samantha L., Welfer, Griffin A., Freudenthal, Bret D., Opresko, Patricia L.

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Complement C3-Targeted Gene Therapy Restricts Onset and Progression of Neurodegeneration in Chronic Mouse Glaucoma von Bosco, Alejandra, Anderson, Sarah R., Breen, Kevin T., Romero, Cesar O., Steele, Michael R., Chiodo, Vince A., Boye, Sanford L., Hauswirth, William W., Tomlinson, Stephen, Vetter, Monica L.

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Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors von Kay, Christine N, Ryals, Renee C, Aslanidi, George V, Min, Seok Hong, Ruan, Qing, Sun, Jingfen, Dyka, Frank M, Kasuga, Daniel, Ayala, Andrea E, Van Vliet, Kim, Agbandje-McKenna, Mavis, Hauswirth, William W, Boye, Sanford L, Boye, Shannon E

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Improving retinal vascular endothelial cell tropism through rational rAAV capsid design von Periasamy, Ramesh, Patel, Dwani D, Boye, Sanford L, Boye, Shannon E, Lipinski, Daniel M

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Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a FLT3 Mutation von Stone, Richard M, Mandrekar, Sumithra J, Sanford, Ben L, Laumann, Kristina, Geyer, Susan, Bloomfield, Clara D, Thiede, Christian, Prior, Thomas W, Döhner, Konstanze, Marcucci, Guido, Lo-Coco, Francesco, Klisovic, Rebecca B, Wei, Andrew, Sierra, Jorge, Sanz, Miguel A, Brandwein, Joseph M, de Witte, Theo, Niederwieser, Dietger, Appelbaum, Frederick R, Medeiros, Bruno C, Tallman, Martin S, Krauter, Jürgen, Schlenk, Richard F, Ganser, Arnold, Serve, Hubert, Ehninger, Gerhard, Amadori, Sergio, Larson, Richard A, Döhner, Hartmut

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Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial von Ghazi, Nicola G., Abboud, Emad B., Nowilaty, Sawsan R., Alkuraya, Hisham, Alhommadi, Abdulrahman, Cai, Huimin, Hou, Rui, Deng, Wen-Tao, Boye, Sanford L., Almaghamsi, Abdulrahman, Al Saikhan, Fahad, Al-Dhibi, Hassan, Birch, David, Chung, Christopher, Colak, Dilek, LaVail, Matthew M., Vollrath, Douglas, Erger, Kirsten, Wang, Wenqiu, Conlon, Thomas, Zhang, Kang, Hauswirth, William, Alkuraya, Fowzan S.

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Observations on Real-Time Prostate Gland Motion Using Electromagnetic Tracking von Langen, Katja M., Ph.D, Willoughby, Twyla R., M.S, Meeks, Sanford L., Ph.D, Santhanam, Anand, Ph.D, Cunningham, Alexis, B.A, Levine, Lisa, Ph.D, Kupelian, Patrick A., M.D

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A Comprehensive Review of Retinal Gene Therapy von Boye, Shannon E, Boye, Sanford L, Lewin, Alfred S, Hauswirth, William W

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Impact of Heparan Sulfate Binding on Transduction of Retina by Recombinant Adeno-Associated Virus Vectors von Boye, Sanford L, Bennett, Antonette, Scalabrino, Miranda L, McCullough, K Tyler, Van Vliet, Kim, Choudhury, Shreyasi, Ruan, Qing, Peterson, James, Agbandje-McKenna, Mavis, Boye, Shannon E

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Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber’s hereditary optic neuropathy in a mouse model von Yu, Hong, Koilkonda, Rajeshwari D, Chou, Tsung-Han, Porciatti, Vittorio, Ozdemir, Sacide S, Chiodo, Vince, Boye, Sanford L, Boye, Shannon E, Hauswirth, William W, Lewin, Alfred S, Guy, John

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Functional genomic screening identifies dual leucine zipper kinase as a key mediator of retinal ganglion cell death von Welsbie, Derek S., Yang, Zhiyong, Ge, Yan, Mitchell, Katherine L., Zhou, Xinrong, Martin, Scott E., Berlinicke, Cynthia A., Hackler, Laszlo, Fuller, John, Fu, Jie, Cao, Li-hui, Han, Bing, Auld, Douglas, Xue, Tian, Hirai, Syu-ichi, Germain, Lucie, Simard-Bisson, Caroline, Blouin, Richard, Nguyen, Judy V., Davis, Chung-ha O., Enke, Raymond A., Boye, Sanford L., Merbs, Shannath L., Marsh-Armstrong, Nicholas, Hauswirth, William W., DiAntonio, Aaron, Nickells, Robert W., Inglese, James, Hanes, Justin, Yau, King-Wai, Quigley, Harry A., Zack, Donald J.

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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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SARM1 depletion rescues NMNAT1-dependent photoreceptor cell death and retinal degeneration von Sasaki, Yo, Kakita, Hiroki, Kubota, Shunsuke, Sene, Abdoulaye, Lee, Tae Jun, Ban, Norimitsu, Dong, Zhenyu, Lin, Joseph B, Boye, Sanford L, DiAntonio, Aaron, Boye, Shannon E, Apte, Rajendra S, Milbrandt, Jeffrey

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Promotion of endoplasmic reticulum retrotranslocation by overexpression of E3 ubiquitin‐protein ligase synoviolin 1 reduces endoplasmic reticulum stress and preserves cone photorec... von Yang, Fan, Ma, Hongwei, Boye, Sanford L., Boye, Shannon E., Ding, Xi‐Qin

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BG4 antibody can recognize telomeric G-quadruplexes harboring destabilizing base modifications and lesions von Johnson, Samuel A, Paul, Tapas, Sanford, Samantha L, Schnable, Brittani L, Detwiler, Ariana C, Thosar, Sanjana A, Van Houten, Bennett, Myong, Sua, Opresko, Patricia L

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We need to be braver about the generalizability crisis von Braver, Todd S., Braver, Sanford L.

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