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Expert Opinion on Biological Therapy Volume 11, 2011 - Issue 3
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Drug Evaluations

Clinical gene therapy for the treatment of RPE65-associated Leber congenital amaurosis

Linda Stein University of Massachusetts Medical School, UMass Memorial Eye Center, Department of Ophthalmology, 281 Lincoln St., Worcester, MA 01605, USA +508 334 9072; +508 334 4655; [email protected]
, MS,
Kamolika Roy University of Massachusetts Medical School, UMass Memorial Eye Center, Department of Ophthalmology, 281 Lincoln St., Worcester, MA 01605, USA +508 334 9072; +508 334 4655; [email protected]
, BS,
Lei Lei University of Massachusetts Medical School, UMass Memorial Eye Center, Department of Ophthalmology, 281 Lincoln St., Worcester, MA 01605, USA +508 334 9072; +508 334 4655; [email protected]
, BS &
Shalesh Kaushal University of Massachusetts Medical School, UMass Memorial Eye Center, Department of Ophthalmology, 281 Lincoln St., Worcester, MA 01605, USA +508 334 9072; +508 334 4655; [email protected]
, MD PhD
Pages 429-439 | Published online: 08 Feb 2011

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W. Bailey Glen$suffix/text()$suffix/text(), M. Millicent W. Peterseim, Ramses Badilla, Iya Znoyko, Andre Bourg, Robert Wilson, Gary Hardiman, Daynna Wolff & Joaquin Martinez. (2019) A high prevalence of biallelic RPE65 mutations in Costa Rican children with Leber congenital amaurosis and early-onset retinal dystrophy. Ophthalmic Genetics 40:2, pages 110-117.
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Zhen Yi, Christina Zeitz, Takeshi Iwata, J. Fielding Hejtmancik & Qingjiong Zhang. 2021. Advances in Vision Research, Volume III. Advances in Vision Research, Volume III 181 196 .
Luke A. WileyErin R. BurnightEmily E. KaalbergChunhua JiaoMegan J. RikerJennifer A. HalderMeagan A. LuseIan C. HanStephen R. RussellElliott H. SohnEdwin M. StoneBudd A. TuckerRobert F. Mullins. (2018) Assessment of Adeno-Associated Virus Serotype Tropism in Human Retinal Explants. Human Gene Therapy 29:4, pages 424-436.
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Wenhua Zheng, Qian Meng, Haitao Wang, Fengxia Yan, Peter J. Little, Xinguo Deng & Shaofen Lin. (2017) IGF-1-Mediated Survival from Induced Death of Human Primary Cultured Retinal Pigment Epithelial Cells Is Mediated by an Akt-Dependent Signaling Pathway. Molecular Neurobiology 55:3, pages 1915-1927.
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Luke A. WileyErin R. BurnightArlene V. DrackBailey B. BanachDalyz OchoaCathryn M. CranstonRobert A. MadumbaJade S. EastRobert F. MullinsEdwin M. StoneBudd A. Tucker. (2016) Using Patient-Specific Induced Pluripotent Stem Cells and Wild-Type Mice to Develop a Gene Augmentation-Based Strategy to Treat CLN3 -Associated Retinal Degeneration . Human Gene Therapy 27:10, pages 835-846.
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Yuhong Wang, Ammaji Rajala & Raju Rajala. (2015) Lipid Nanoparticles for Ocular Gene Delivery. Journal of Functional Biomaterials 6:2, pages 379-394.
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Alexander Bello, Allan Chand, Jenna Aviles, Geoff Soule, Alberto Auricchio & Gary P. Kobinger. (2014) Novel Adeno-associated Viruses Derived From Pig Tissues Transduce Most Major Organs in Mice. Scientific Reports 4:1.
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Andrew C. Weitz & James D. Weiland. 2014. Neural Computation, Neural Devices, and Neural Prosthesis. Neural Computation, Neural Devices, and Neural Prosthesis 157 188 .
Mark S. Humayun, Rodrigo A. Brant Fernandes & James D. Weiland. 2013. Retina. Retina 2078 2093 .
Julia Reetz, Ottmar Herchenröder, Anke Schmidt & Brigitte M. Pützer. 2013. Regenerative Medicine. Regenerative Medicine 475 503 .
Pasqualina Colella & Alberto Auricchio. 2013. Tissue-Protective Cytokines. Tissue-Protective Cytokines 237 263 .
Ritobrato Datta, Jongho Lee, Jeffrey Duda, Brian B. Avants, Charles H. Vite, Ben Tseng, James C. Gee, Gustavo D. Aguirre & Geoffrey K. Aguirre. (2012) A Digital Atlas of the Dog Brain. PLoS ONE 7:12, pages e52140.
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Alice F. TarantalSonia I. Skarlatos. (2012) Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases: An NHLBI Resource for the Gene Therapy Community. Human Gene Therapy 23:11, pages 1130-1135.
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Ping-Jie Xiao, Thomas B Lentz & R Jude Samulski. (2012) Recombinant adeno-associated virus: clinical application and development as a gene-therapy vector. Therapeutic Delivery 3:7, pages 835-856.
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Huibi Cao, Robert S. Molday & Jim Hu. (2012) Gene therapy: light is finally in the tunnel. Protein & Cell 2:12, pages 973-989.
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Teresa Borrás. (2011) Gene therapy strategies in glaucoma and application for steroid-induced hypertension. Saudi Journal of Ophthalmology 25:4, pages 353-362.
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