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Select item 64413581.

Seven novel variants expand the spectrum of RPE65-related Leber congenital amaurosis in the Chinese population

Zilin Zhong, Feng Rong, Yinghui Dai, Alakezi Yibulayin, Lin Zeng, Jian Liao, Liefeng Wang, Zhihua Huang, Zhenping Zhou, Jianjun Chen

Mol Vis. 2019; 25: 204–214. Published online 2019 Mar 18.

PMCID:: PMC6441358

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Select item 57630292.

Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis

Guylène Le Meur, Pierre Lebranchu, Fanny Billaud, Oumeya Adjali, Sébastien Schmitt, Stéphane Bézieau, Yann Péréon, Romain Valabregue, Catherine Ivan, Christophe Darmon, Philippe Moullier, Fabienne Rolling, Michel Weber

Mol Ther. 2018 Jan 3; 26(1): 256–268. Published online 2017 Sep 19. doi: 10.1016/j.ymthe.2017.09.014

PMCID:: PMC5763029

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Select item 36741123.

Three Year Follow-Up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2

Francesco Testa, Albert M Maguire, Settimio Rossi, Eric A Pierce, Paolo Melillo, Kathleen Marshall, Sandro Banfi, Enrico M. Surace, Junwei Sun, Carmela Acerra, J. Fraser Wright, Jennifer Wellman, Katherine A High, Alberto Auricchio, Jean Bennett, Francesca Simonelli

Ophthalmology. Author manuscript; available in PMC 2014 Jun 1.

Published in final edited form as: Ophthalmology. 2013 Jun; 120(6): 1283–1291. Published online 2013 Mar 6. doi: 10.1016/j.ophtha.2012.11.048

PMCID:: PMC3674112

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Select item 97895264.

Quantitative and Qualitative Characterization of Retinal Dystrophies in Canine Models of Inherited Retinal Diseases Using Spectral Domain Optical Coherence Tomography (SD-OCT)

Shin Ae Park, Jamie Rhodes, Simone Iwabe, Gui-Shuang Ying, Wei Pan, Jiayan Huang, András M. Komáromy

Exp Eye Res. Author manuscript; available in PMC 2023 Jul 1.

Published in final edited form as: Exp Eye Res. 2022 Jul; 220: 109106. Published online 2022 May 16. doi: 10.1016/j.exer.2022.109106

PMCID:: PMC9789526

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Select item 95551125.

Familial risk associated with lung cancer as a second primary malignancy in first-degree relatives

Jianguang Ji, Jan Sundquist, Kristina Sundquist, Guoqiao Zheng

BMC Cancer. 2022; 22: 1057. Published online 2022 Oct 12. doi: 10.1186/s12885-022-10149-7

PMCID:: PMC9555112

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Select item 73518776.

Perspectives on Gene Therapy: Choroideremia Represents a Challenging Model for the Treatment of Other Inherited Retinal Degenerations

Ian M. MacDonald, Christopher Moen, Jacque L. Duncan, Stephen H. Tsang, Jasmina Cehajic-Kapetanovic, Tomas S. Aleman

Transl Vis Sci Technol. 2020 Feb; 9(3): 17. Published online 2020 Feb 14. doi: 10.1167/tvst.9.3.17

PMCID:: PMC7351877

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Select item 71482657.

Impact of gene therapy for canine monogenic diseases on the progress of preclinical studies

Marek Switonski

J Appl Genet. 2020; 61(2): 179–186. Published online 2020 Mar 18. doi: 10.1007/s13353-020-00554-8

PMCID:: PMC7148265

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Select item 67007488.

SubILM Injection of AAV for Gene Delivery to the Retina

Paul D. Gamlin, John J. Alexander, Sanford L. Boye, C. Douglas Witherspoon, Shannon E. Boye

Methods Mol Biol. Author manuscript; available in PMC 2019 Aug 20.

Published in final edited form as: Methods Mol Biol. 2019; 1950: 249–262. doi: 10.1007/978-1-4939-9139-6_14

PMCID:: PMC6700748

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Select item 65471289.

Deep Scleral Exposure: A Degenerative Outcome of End-Stage Stargardt Disease

WINSTON LEE, JANA ZERNANT, TAKAYUKI NAGASAKI, STEPHEN H. TSANG, RANDO ALLIKMETS

Am J Ophthalmol. Author manuscript; available in PMC 2019 Jun 4.

Published in final edited form as: Am J Ophthalmol. 2018 Nov; 195: 16–25. Published online 2018 Jul 26. doi: 10.1016/j.ajo.2018.07.018

PMCID:: PMC6547128

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Select item 497554910.

CRISPR challenges in treating retinal disease

Micah A. Chrenek, John M. Nickerson, Jeffrey H. Boatright

Asia Pac J Ophthalmol (Phila) Author manuscript; available in PMC 2017 Jul 1.

Published in final edited form as: Asia Pac J Ophthalmol (Phila). 2016 Jul-Aug; 5(4): 304–308. doi: 10.1097/APO.0000000000000225

PMCID:: PMC4975549

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Select item 355968111.

AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina

Luk H. Vandenberghe, Peter Bell, Albert M. Maguire, Ru Xiao, Tim B. Hopkins, Rebecca Grant, Jean Bennett, James M. Wilson

PLoS One. 2013; 8(1): e53463. Published online 2013 Jan 30. doi: 10.1371/journal.pone.0053463

Correction in:: PLoS One. 2013; 8(8): 10.1371/annotation/64b90996-4634-4c63-b737-634397b0b445.

PMCID:: PMC3559681

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Select item 329767312.

RPE65 Is Present in Human Green/Red Cones and Promotes Photopigment Regeneration in an In Vitro Cone Cell Model

Peter H. Tang, Mona C. Buhusi, Jian-Xing Ma, Rosalie K. Crouch

J Neurosci. 2011 Dec 14; 31(50): 18618–18626. doi: 10.1523/JNEUROSCI.4265-11.2011

PMCID:: PMC3297673

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Select item 310477913.

The human visual cortex responds to gene therapy–mediated recovery of retinal function

Manzar Ashtari, Laura L. Cyckowski, Justin F. Monroe, Kathleen A. Marshall, Daniel C. Chung, Alberto Auricchio, Francesca Simonelli, Bart P. Leroy, Albert M. Maguire, Kenneth S. Shindler, Jean Bennett

J Clin Invest. 2011 Jun 1; 121(6): 2160–2168. Published online 2011 May 23. doi: 10.1172/JCI57377

Correction in:: J Clin Invest. 2011 Jul 1; 121(7): 2945.

PMCID:: PMC3104779

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Select item 283944014.

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

Francesca Simonelli, Albert M Maguire, Francesco Testa, Eric A Pierce, Federico Mingozzi, Jeannette L Bennicelli, Settimio Rossi, Kathleen Marshall, Sandro Banfi, Enrico M Surace, Junwei Sun, T Michael Redmond, Xiaosong Zhu, Kenneth S Shindler, Gui-Shuang Ying, Carmela Ziviello, Carmela Acerra, J Fraser Wright, Jennifer Wellman McDonnell, Katherine A High, Jean Bennett, Alberto Auricchio

Mol Ther. 2010 Mar; 18(3): 643–650. Published online 2009 Dec 1. doi: 10.1038/mt.2009.277

PMCID:: PMC2839440

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Select item 282974815.

Safety and Efficacy of Gene Transfer for Leber’s Congenital Amaurosis

Albert M. Maguire, Francesca Simonelli, Eric A. Pierce, Edward N. Pugh, Jr., Federico Mingozzi, Jeannette Bennicelli, Sandro Banfi, Kathleen A. Marshall, Francesco Testa, Enrico M. Surace, Settimio Rossi, Arkady Lyubarsky, Valder R. Arruda, Barbara Konkle, Edwin Stone, Junwei Sun, Jonathan Jacobs, Lou Dell’Osso, Richard Hertle, Jian-xing Ma, T. Michael Redmond, Xiaosong Zhu, Bernd Hauck, Olga Zelenaia, Kenneth S. Shindler, Maureen G. Maguire, J. Fraser Wright, Nicholas J. Volpe, Jennifer Wellman McDonnell, Alberto Auricchio, Katherine A. High, Jean Bennett

N Engl J Med. Author manuscript; available in PMC 2010 Mar 1.

Published in final edited form as: N Engl J Med. 2008 May 22; 358(21): 2240–2248. Published online 2008 Apr 27. doi: 10.1056/NEJMoa0802315

PMCID:: PMC2829748

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Select item 270120816.

Catalytic Properties of Botulinum Neurotoxins Subtypes A3 and A4

James S. Henkel, Mark Jacobson, William Tepp, Christina Pier, Eric A. Johnson, Joseph T. Barbieri

Biochemistry. Author manuscript; available in PMC 2010 Mar 24.

Published in final edited form as: Biochemistry. 2009 Mar 24; 48(11): 2522–2528. doi: 10.1021/bi801686b

PMCID:: PMC2701208

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Select item 157245417.

Evidence from immunoneutralization and antisense studies that the inhibitory actions of glucocorticoids on growth hormone release in vitro require annexin 1 (lipocortin 1)

A D Taylor, H C Christian, J F Morris, R J Flower, J C Buckingham

Br J Pharmacol. 2000 Dec; 131(7): 1309–1316. doi: 10.1038/sj.bjp.0703694

PMCID:: PMC1572454

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Select item 941487918.

Lentiviral Vectors for Ocular Gene Therapy

Yvan Arsenijevic, Adeline Berger, Florian Udry, Corinne Kostic

Pharmaceutics. 2022 Aug; 14(8): 1605. Published online 2022 Jul 31. doi: 10.3390/pharmaceutics14081605

PMCID:: PMC9414879

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Select item 868276119.

Assessing Photoreceptor Status in Retinal Dystrophies: From High-Resolution Imaging to Functional Vision

José-Alain Sahel, Kate Grieve, Chloé Pagot, Colas Authié, Saddek Mohand-Said, Michel Paques, Isabelle Audo, Karine Becker, Anne-Elisabeth Chaumet-Riffaud, Line Azoulay, Emmanuel Gutman, Thierry Léveillard, Christina Zeitz, Serge Picaud, Deniz Dalkara, Katia Marazova

Am J Ophthalmol. 2021 Oct; 230: 12–47. doi: 10.1016/j.ajo.2021.04.013

PMCID:: PMC8682761

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Select item 851718420.

Toward the Treatment of Inherited Diseases of the Retina Using CRISPR-Based Gene Editing

Jennifer Hernández-Juárez, Genaro Rodríguez-Uribe, Shyamanga Borooah

Front Med (Lausanne) 2021; 8: 698521. Published online 2021 Oct 1. doi: 10.3389/fmed.2021.698521

PMCID:: PMC8517184

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