Works by Gray, Steven J.

Results: 38

Thionylimido Complexes.

Published in:

Zeitschrift für Anorganische und Allgemeine Chemie, 2020, v. 646, n. 22, p. 1795, doi. 10.1002/zaac.202000238

By:

McGeachie, Liam J. R.;
Carpenter‐Warren, Cameron L.;
Cordes, David B.;
Bühl, Michael;
Gray, Steven J.;
Hua, Guoxiong;
Slawin, Alexandra M. Z.;
Woollins, J. Derek

Publication type:

Article

Targeted muscle reinnervation to improve electromyography signals for advanced myoelectric prosthetic limbs: a series of seven patients.

Published in:

ANZ Journal of Surgery, 2020, v. 90, n. 4, p. 591, doi. 10.1111/ans.15664

By:

Myers, Harley;
Lu, David;
Gray, Steven J.;
Bruscino‐Raiola, Frank

Publication type:

Article

Optical coherence tomography features in brothers with aspartylglucosaminuria.

Published in:

Annals of Clinical & Translational Neurology, 2018, v. 5, n. 12, p. 1622, doi. 10.1002/acn3.672

By:

Goodspeed, Kimberly;
Harder, Lana;
Hughes, Samuel;
Conger, Darrel;
Taravella, Julia;
Gray, Steven J;
Minassian, Berge

Publication type:

Article

RNAi Knockdown of EHMT2 in Maternal Expression of Prader–Willi Syndrome Genes.

Published in:

Genes, 2024, v. 15, n. 11, p. 1366, doi. 10.3390/genes15111366

By:

Zaric, Violeta;
Kang, Hye Ri;
Rybalchenko, Volodymyr;
Zigman, Jeffrey M.;
Gray, Steven J.;
Butler, Ryan K.

Publication type:

Article

The Efficacy of a Human-Ready mini MECP2 Gene Therapy in a Pre-Clinical Model of Rett Syndrome.

Published in:

Genes, 2024, v. 15, n. 1, p. 31, doi. 10.3390/genes15010031

By:

Sadhu, Chanchal;
Lyons, Christopher;
Oh, Jiyoung;
Jagadeeswaran, Indumathy;
Gray, Steven J.;
Sinnett, Sarah E.

Publication type:

Article

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

Published in:

Genes, 2023, v. 14, n. 8, p. 1660, doi. 10.3390/genes14081660

By:

Casy, Widler;
Garza, Irvin T.;
Chen, Xin;
Dong, Thomas;
Hu, Yuhui;
Kanchwala, Mohammed;
Trygg, Cynthia B.;
Shyng, Charles;
Xing, Chao;
Bunnell, Bruce A.;
Braun, Stephen E.;
Gray, Steven J.

Publication type:

Article

Correction: Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation.

Published in:

2018

By:

Ramsingh, Arlene I.;
Gray, Steven J.;
Reilly, Andrew;
Koday, Michael;
Bratt, Debbie;
Koday, Merika Treants;
Munson, Paul;
Murnane, Robert;
Smedley, Jeremy;
Hu, Yuhui;
Messer, Anne;
Fuller, Deborah Heydenburg

Publication type:

Correction Notice

Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation.

Published in:

PLoS ONE, 2018, v. 13, n. 6, p. 1, doi. 10.1371/journal.pone.0198154

By:

Ramsingh, Arlene I.;
Gray, Steven J.;
Reilly, Andrew;
Koday, Michael;
Bratt, Debbie;
Koday, Merika Treants;
Murnane, Robert;
Smedley, Jeremy;
Hu, Yuhui;
Messer, Anne;
Fuller, Deborah Heydenburg

Publication type:

Article

Immunological considerations for treating globoid cell leukodystrophy.

Published in:

Journal of Neuroscience Research, 2016, v. 94, n. 11, p. 1349, doi. 10.1002/jnr.23874

By:

Karumuthil‐Melethil, Subha;
Gray, Steven J.

Publication type:

Article

Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant.

Published in:

Journal of Neuroscience Research, 2016, v. 94, n. 11, p. 1138, doi. 10.1002/jnr.23882

By:

Karumuthil‐Melethil, Subha;
Marshall, Michael S.;
Heindel, Clifford;
Jakubauskas, Benas;
Bongarzone, Ernesto R.;
Gray, Steven J.

Publication type:

Article

Advancing the pathologic phenotype of giant axonal neuropathy: early involvement of the ocular lens.

Published in:

2019

By:

Armao, Diane;
Bouldin, Thomas W.;
Bailey, Rachel M.;
Hooper, Jody E.;
Bharucha, Diana X.;
Gray, Steven J.

Publication type:

Letter

Efficacy of dual intracerebroventricular and intravitreal CLN5 gene therapy in sheep prompts the first clinical trial to treat CLN5 Batten disease.

Published in:

Frontiers in Pharmacology, 2023, p. 1, doi. 10.3389/fphar.2023.1212235

By:

Murray, Samantha J.;
Wellby, Martin P.;
Barrell, Graham K.;
Russell, Katharina N.;
Deane, Ashley R.;
Wynyard, John R.;
Gray, Steven J.;
Palmer, David N.;
Mitchell, Nadia L.

Publication type:

Article

Efficacy of dual intracerebroventricular and intravitreal CLN5 gene therapy in sheep prompts the first clinical trial to treat CLN5 Batten disease.

Published in:

Frontiers in Pharmacology, 2023, p. 1, doi. 10.3389/fphar.2023.1212235

By:

Murray, Samantha J.;
Wellby, Martin P.;
Barrell, Graham K.;
Russell, Katharina N.;
Deane, Ashley R.;
Wynyard, John R.;
Gray, Steven J.;
Palmer, David N.;
Mitchell, Nadia L.

Publication type:

Article

Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten disease.

Published in:

Frontiers in Genetics, 2023, p. 1, doi. 10.3389/fgene.2023.1212228

By:

Mitchell, Nadia L.;
Murray, Samantha J.;
Wellby, Martin P.;
Barrell, Graham K.;
Russell, Katharina N.;
Deane, Ashley R.;
Wynyard, John R.;
Palmer, Madeleine J.;
Pulickan, Anila;
Prendergast, Phillipa M.;
Casy, Widler;
Gray, Steven J.;
Palmer, David N.

Publication type:

Article

In Cellulo Examination of a Beta-Alpha Hybrid Construct of Beta-Hexosaminidase A Subunits, Reported to Interact with the GM2 Activator Protein and Hydrolyze GM2 Ganglioside.

Published in:

PLoS ONE, 2013, v. 8, n. 3, p. 1, doi. 10.1371/journal.pone.0057908

By:

Sinici, Incilay;
Yonekawa, Sayuri;
Tkachyova, Ilona;
Gray, Steven J.;
Samulski, R. Jude;
Wakarchuk, Warren;
Mark, Brian L.;
Mahuran, Don J.

Publication type:

Article

Extensive rod and cone photoreceptor-cell degeneration in rat models of giant axonal neuropathy: implications for gene therapy of human disease.

Published in:

Ophthalmic Genetics, 2021, v. 42, n. 5, p. 600, doi. 10.1080/13816810.2021.1923036

By:

Armao, Diane;
Bouldin, Thomas W.;
Bailey, Rachel M.;
Gray, Steven J.

Publication type:

Article

Intermediate filament protein accumulation in motor neurons derived from giant axonal neuropathy iPSCs rescued by restoration of gigaxonin.

Published in:

Human Molecular Genetics, 2015, v. 24, n. 5, p. 1420, doi. 10.1093/hmg/ddu556

By:

Johnson-Kerner, Bethany L.;
Ahmad, Faizzan S.;
Diaz, Alejandro Garcia;
Greene, John Palmer;
Gray, Steven J.;
Samulski, Richard Jude;
Chung, Wendy K.;
Van Coster, Rudy;
Maertens, Paul;
Noggle, Scott A.;
Henderson, Christopher E.;
Wichterle, Hynek

Publication type:

Article

SURF1 Deficiency: Expanding on Disease Phenotype and Assessing Disease Burden by Describing Clinical and Biochemical Phenotype.

Published in:

American Journal of Medical Genetics. Part A, 2025, v. 197, n. 4, p. 1, doi. 10.1002/ajmg.a.63947

By:

Kayani, Saima;
Daescu, Victor;
Dahshi, Hamza;
Messahel, Souad;
Woleban, Kasey;
Minassian, Berge A.;
Ling, Qinglan;
Gray, Steven J.

Publication type:

Article

Giant axonal neuropathy: cross-sectional analysis of a large natural history cohort.

Published in:

2021

By:

Bharucha-Goebel, Diana X;
Norato, Gina;
Saade, Dimah;
Paredes, Eduardo;
Biancavilla, Victoria;
Donkervoort, Sandra;
Kaur, Rupleen;
Lehky, Tanya;
Fink, Margaret;
Armao, Diane;
Gray, Steven J;
Waite, Melissa;
Debs, Sarah;
Averion, Gilberto;
Hu, Ying;
Zein, Wadih M;
Foley, A Reghan;
Jain, Minal;
Bönnemann, Carsten G

Publication type:

journal article

Engineered microRNA-based regulatory element permits safe high-dose miniMECP2 gene therapy in Rett mice.

Published in:

2021

By:

Sinnett, Sarah E;
Boyle, Emily;
Lyons, Christopher;
Gray, Steven J

Publication type:

journal article

Mechanism-Based Combination Treatment Dramatically Increases Therapeutic Efficacy in Murine Globoid Cell Leukodystrophy.

Published in:

Journal of Neuroscience, 2015, p. 6495, doi. 10.1523/JNEUROSCI.4199-14.2015

By:

Hawkins-Salsbury, Jacqueline A.;
Shea, Lauren;
Xuntian Jiang;
Hunter, Daniel A.;
Guzman, A. Miguel;
Reddy, Adarsh S.;
Qin, Elizabeth Y.;
Li, Yedda;
Gray, Steven J.;
Ory, Daniel S.;
Sands, Mark S.

Publication type:

Article

Novel oligodendroglial alpha synuclein viral vector models of multiple system atrophy: studies in rodents and nonhuman primates.

Published in:

Acta Neuropathologica Communications, 2017, v. 5, p. 1, doi. 10.1186/s40478-017-0451-7

By:

Mandel, Ronald J.;
Marmion, David J.;
Kirik, Deniz;
Yaping Chu;
Heindel, Clifford;
McCown, Thomas;
Gray, Steven J.;
Kordower, Jeffrey H.

Publication type:

Article

Identification of Small Molecule Compounds for Pharmacological Chaperone Therapy of Aspartylglucosaminuria.

Published in:

Scientific Reports, 2016, p. 37583, doi. 10.1038/srep37583

By:

Banning, Antje;
Gülec, Christina;
Rouvinen, Juha;
Gray, Steven J.;
Tikkanen, Ritva

Publication type:

Article

An Origin of DNA Replication in the Promoter Region of the Human Fragile X Mental Retardation (FMR1) Gene.

Published in:

Molecular & Cellular Biology, 2007, v. 27, n. 2, p. 2, doi. 10.1128/MCB.01382-06

By:

Gray, Steven J.;
Gerhardt, Jeannine;
Doerfler, Walter;
Small, Lawrence E.;
Fanning, Ellen

Publication type:

Article

AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model.

Published in:

2021

By:

Presa, Maximiliano;
Bailey, Rachel M.;
Davis, Crystal;
Murphy, Tara;
Cook, Jenn;
Walls, Randy;
Wilpan, Hannah;
Bogdanik, Laurent;
Lenk, Guy M.;
Burgess, Robert W.;
Gray, Steven J.;
Lutz, Cathleen

Publication type:

journal article

Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.

Published in:

2020

By:

Bradbury, Allison M.;
Bagel, Jessica H.;
Nguyen, Duc;
Lykken, Erik A.;
Salvador, Jill Pesayco;
Xuntian Jiang;
Swain, Gary P.;
Assenmacher, Charles A.;
Hendricks, Ian J.;
Miyadera, Keiko;
Hess, Rebecka S.;
Ostrager, Arielle;
ODonnell, Patricia;
Sands, Mark S.;
Ory, Daniel S.;
Shelton, G. Diane;
Bongarzone, Ernesto R.;
Gray, Steven J.;
Vite, Charles H.;
Pesayco Salvador, Jill

Publication type:

journal article

Improved Survival and Reduced Phenotypic Severity Following AAV9/MECP2 Gene Transfer to Neonatal and Juvenile Male Mecp2 Knockout Mice.

Published in:

Molecular Therapy, 2013, v. 21, n. 1, p. 18, doi. 10.1038/mt.2012.200

By:

Gadalla, Kamal KE;
Bailey, Mark ES;
Spike, Rosemary C;
Ross, Paul D;
Woodard, Kenton T;
Kalburgi, Sahana Nagabhushan;
Bachaboina, Lavanya;
Deng, Jie V;
West, Anne E;
Samulski, R Jude;
Gray, Steven J;
Cobb, Stuart R

Publication type:

Article

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector.

Published in:

Molecular Therapy, 2012, v. 20, n. 2, p. 443, doi. 10.1038/mt.2011.237

By:

Bowles, Dawn E;
McPhee, Scott WJ;
Li, Chengwen;
Gray, Steven J;
Samulski, Jade J;
Camp, Angelique S;
Li, Juan;
Wang, Bing;
Monahan, Paul E;
Rabinowitz, Joseph E;
Grieger, Joshua C;
Govindasamy, Lakshmanan;
Agbandje-McKenna, Mavis;
Xiao, Xiao;
Samulski, R Jude

Publication type:

Article

Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates.

Published in:

Molecular Therapy, 2011, v. 19, n. 6, p. 1058, doi. 10.1038/mt.2011.72

By:

Gray, Steven J;
Matagne, Valerie;
Bachaboina, Lavanya;
Yadav, Swati;
Ojeda, Sergio R;
Samulski, R Jude

Publication type:

Article

Transient Demyelination Increases the Efficiency of Retrograde AAV Transduction.

Published in:

Molecular Therapy, 2010, v. 18, n. 8, p. 1496, doi. 10.1038/mt.2010.97

By:

Hollis II, Edmund R.;
Jamshidi, Pouya;
Lorenzana, Ariana O.;
Lee, Jae K.;
Gray, Steven J.;
Samulski, Richard J.;
Zheng, Binhai;
Tuszynski, Mark H.

Publication type:

Article

Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB).

Published in:

Molecular Therapy, 2010, v. 18, n. 3, p. 570, doi. 10.1038/mt.2009.292

By:

Gray, Steven J.;
Blake, Bonita L.;
Criswell, Hugh E.;
Nicolson, Sarah C.;
Samulski, R. Jude;
McCown, Thomas J.

Publication type:

Article

Comparative outcome analysis of osseointegrated reconstruction and replantation for digital amputations.

Published in:

Journal of Hand Surgery (17531934), 2022, v. 47, n. 5, p. 453, doi. 10.1177/17531934211030752

By:

Li, Andrew T.;
Shepherd, Angela;
Gray, Steven J.;
Slattery, Philip;
Bruscino-Raiola, Frank

Publication type:

Article

Knockout of the CMP–Sialic Acid Transporter SLC35A1 in Human Cell Lines Increases Transduction Efficiency of Adeno-Associated Virus 9: Implications for Gene Therapy Potency Assays.

Published in:

Cells (2073-4409), 2021, v. 10, n. 5, p. 1259, doi. 10.3390/cells10051259

By:

Banning, Antje;
Zakrzewicz, Anna;
Chen, Xin;
Gray, Steven J.;
Tikkanen, Ritva;
Brakebusch, Cord;
d'Azzo, Alessandra

Publication type:

Article

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

Published in:

International Journal of Molecular Sciences, 2023, v. 24, n. 19, p. 14611, doi. 10.3390/ijms241914611

By:

Vyas, Meera;
Deschenes, Natalie M.;
Osmon, Karlaina J. L.;
Chen, Zhilin;
Ahmad, Imtiaz;
Kot, Shalini;
Thompson, Patrick;
Richmond, Chris;
Gray, Steven J.;
Walia, Jagdeep S.

Publication type:

Article

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

Published in:

International Journal of Molecular Sciences, 2023, v. 24, n. 11, p. 9217, doi. 10.3390/ijms24119217

By:

Deschenes, Natalie M.;
Cheng, Camilyn;
Ryckman, Alex E.;
Quinville, Brianna M.;
Khanal, Prem;
Mitchell, Melissa;
Chen, Zhilin;
Sangrar, Waheed;
Gray, Steven J.;
Walia, Jagdeep S.

Publication type:

Article

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

Published in:

International Journal of Molecular Sciences, 2021, v. 22, n. 13, p. 6751, doi. 10.3390/ijms22136751

By:

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

Publication type:

Article

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

Published in:

International Journal of Molecular Sciences, 2017, v. 18, n. 4, p. 706, doi. 10.3390/ijms18040706

By:

Banning, Antje;
König, Jan F.;
Gray, Steven J.;
Tikkanen, Ritva

Publication type:

Article

Riboflavin transporter deficiency: AAV9-SLC52A2 gene therapy as a new therapeutic strategy.

Published in:

Frontiers in Cellular Neuroscience, 2025, p. 1, doi. 10.3389/fncel.2025.1523773

By:

Mei, Cecilia;
Magliocca, Valentina;
Chen, Xin;
Massey, Keith;
Gonzalez-Cordero, Anai;
Gray, Steven J.;
Tartaglia, Marco;
Bertini, Enrico Silvio;
Corti, Stefania;
Compagnucci, Claudia

Publication type:

Article