Timing of Gene Therapy Interventions: The Earlier, the Better

Por Gray, Steven J
Publicado no Mol Ther (2016)

Viral vectors and delivery strategies for CNS gene therapy

Por Gray, Steven J, Woodard, Kenton T, Samulski, R Jude
Publicado no Ther Deliv (2010)

Comparison of High-Dose Intracisterna Magna and Lumbar Puncture Intrathecal Delivery of AAV9 in Mice to Treat Neuropathies

Por Bailey, Rachel M., Rozenberg, Alejandra, Gray, Steven J.
Publicado no Brain Res (2020)

Viral Vectors for Gene Delivery to the Central Nervous System

Por Lentz, Thomas B., Gray, Steven J., Samulski, R. Jude
Publicado em 2011

Recent Gene Therapy Advancements for Neurological Diseases

Por Kalburgi, Sahana Nagabhushan, Khan, Nadia N., Gray, Steven J.
Publicado no Discov Med (2013)

Clinical Applications Involving CNS Gene Transfer

Por Kantor, Boris, McCown, Thomas, Leone, Paola, Gray, Steven J.
Publicado no Adv Genet (2014)

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

Por Banning, Antje, König, Jan F., Gray, Steven J., Tikkanen, Ritva
Publicado no Int J Mol Sci (2017)

Identification of Small Molecule Compounds for Pharmacological Chaperone Therapy of Aspartylglucosaminuria

Por Banning, Antje, Gülec, Christina, Rouvinen, Juha, Gray, Steven J., Tikkanen, Ritva
Publicado no Sci Rep (2016)

Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy

Por Bailey, Rachel M., Armao, Diane, Nagabhushan Kalburgi, Sahana, Gray, Steven J.
Publicado no Mol Ther Methods Clin Dev (2018)

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

Por Gray, Steven J., Gerhardt, Jeannine, Doerfler, Walter, Small, Lawrence E., Fanning, Ellen
Publicado em 2007

Discrete Functional Elements Required for Initiation Activity of the Chinese Hamster Dihydrofolate Reductase Origin Beta at Ectopic Chromosomal Sites

Por Gray, Steven J., Liu, Guoqi, Altman, Amy L., Small, Lawrence E., Fanning, Ellen
Publicado em 2006

Global CNS Gene Delivery and Evasion of Anti-AAV Neutralizing Antibodies by Intrathecal AAV Administration in Non-Human Primates

Por Gray, Steven J., Kalburgi, Sahana Nagabhushan, McCown, Thomas J., Samulski, R. Jude
Publicado em 2013

Methods for Gene Transfer to the Central Nervous System

Por Kantor, Boris, Bailey, Rachel M., Wimberly, Keon, Kalburgi, Sahana N., Gray, Steven J.
Publicado no Adv Genet (2014)

Autonomic Nervous System Involvement in the Giant Axonal Neuropathy (GAN) KO Mouse: Implications for Human Disease

Por Armao, Diane, Bailey, Rachel M., Bouldin, Thomas W., Kim, Yongbaek, Gray, Steven J.
Publicado no Clin Auton Res (2016)

Intrathecal Administration of AAV/GALC Vectors in 10-11 Day old Twitcher Mice Improves Survival and is Enhanced by Bone Marrow Transplant

Por Karumuthil-Melethil, Subha, Marshall, Michael S., Heindel, Clifford, Jakubauskas, Benas, Bongarzone, Ernesto, Gray, Steven J.
Publicado no J Neurosci Res (2016)

Reducing the Risk of Adeno-Associated Virus (AAV) Vector Mobilization with AAV Type 5 Vectors

Por Hewitt, F. Curtis, Li, Chengwen, Gray, Steven J., Cockrell, Shelley, Washburn, Michael, Samulski, R. Jude
Publicado em 2009

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

Por Gray, Steven J, Matagne, Valerie, Bachaboina, Lavanya, Yadav, Swati, Ojeda, Sergio R, Samulski, R Jude
Publicado em 2011

Optical coherence tomography features in brothers with aspartylglucosaminuria

Por Goodspeed, Kimberly, Harder, Lana, Hughes, Samuel, Conger, Darrel, Taravella, Julia, Gray, Steven J, Minassian, Berge
Publicado no Ann Clin Transl Neurol (2018)

Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation

Por Chen, Xin, Snanoudj-Verber, Sarah, Pollard, Laura, Hu, Yuhui, Cathey, Sara S., Tikkanen, Ritva, Gray, Steven J.
Publicado no Mol Ther (2021)

Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model

Por Woodley, Evan, Osmon, Karlaina J.L., Thompson, Patrick, Richmond, Christopher, Chen, Zhilin, Gray, Steven J., Walia, Jagdeep S.
Publicado no Mol Ther Methods Clin Dev (2018)