Steven J. Gray - Dissemin

All papers authored by Steven J. Gray

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2023 Chanchal Sadhu, Christopher Lyons, Jiyoung Oh, Indumathy Jagadeeswaran, Steven J. Gray

, Sarah E. Sinnett

The Efficacy of a Human-Ready miniMECP2 Gene Therapy in a Pre-Clinical Model of Rett Syndrome

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Emrah Gumusgoz, Sahba Kasiri, Mayank Verma, Jun Wu, Daniel Villarreal Acha, Ummay Marriam, Sharyl Fyffe-Maricich, Amy Lin, Xin Chen

, Steven J. Gray

, Berge A. Minassian

CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy

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Meera Vyas, Natalie M. Deschenes

, Karlaina J. L. Osmon, Zhilin Chen, Imtiaz Ahmad, Shalini Kot, Patrick Thompson, Chris Richmond, Steven J. Gray

, Jagdeep S. Walia

Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis

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Qinglan Ling, Jessica A. Herstine

, Allison Bradbury, Steven J. Gray

AAV-based in vivo gene therapy for neurological disorders

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Widler Casy, Irvin Garza

, Xin Chen

, Thomas Dong, Yuhui Hu, Mohammed Kanchwala

, Cynthia Trygg, Charles Shyng, Chao Xing

, Bruce Bunnell

, Stephen Braun, Steven Gray

SMRT Sequencing Enables High-Throughput Identification of Novel AAVs from Capsid Shuffling and Directed Evolution

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Hayes Wong, Alexander W. M. Hooper, Hye Ri Kang, Shiron J. Lee

, Jiayi Zhao, Chanchal Sadhu, Satinder Rawat, Steven J. Gray

, David R. Hampson

CNS-dominant human FMRP isoform rescues seizures, fear, and sleep abnormalities in Fmr1-KO mice

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Natalie M. Deschenes

, Camilyn Cheng, Alex E. Ryckman, Brianna M. Quinville, Prem Khanal, Melissa Mitchell, Zhilin Chen, Waheed Sangrar, Steven J. Gray

, Jagdeep S. Walia

Biochemical Correction of GM2 Ganglioside Accumulation in AB-Variant GM2 Gangliosidosis

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Xin Chen, Daniel A. Lim, Michael W. Lawlor, David Dimmock, Charles H. Vite, Thomas Lester, Fatemeh Tavakkoli, Chanchal Sadhu, Suyash Prasad, Steven J. Gray

Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration

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2022 Xin Chen, Thomas Dong, Yuhui Hu, Frances C. Shaffo, Nandkishore R. Belur

, Joseph R. Mazzulli

, Steven J. Gray

AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease

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2021 Qinglan Ling

, Matthew Rioux

, Yuhui Hu, MinJae Lee

, Steven J. Gray

Adeno-associated viral vector serotype 9-based gene replacement therapy for SURF1-related Leigh syndrome

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Shalini Kot

, Subha Karumuthil-Melethil, Evan Woodley, Violeta Zaric, Patrick Thompson, Zhilin Chen, Erik Lykken, John G. Keimel, William F. Kaemmerer, Steven J. Gray

, Jagdeep S. Walia

Investigating Immune Responses to the scAAV9-HEXM Gene Therapy Treatment in Tay–Sachs Disease and Sandhoff Disease Mouse Models

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2017 Antje Banning, Jan F. König, Steven J. Gray

, Ritva Tikkanen

Functional Analysis of the Ser149/Thr149 Variants of Human Aspartylglucosaminidase and Optimization of the Coding Sequence for Protein Production

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All papers authored by Steven J. Gray