Timing of Gene Therapy Interventions: The Earlier, the Better

由 Gray, Steven J
發表在 Mol Ther (2016)

Viral vectors and delivery strategies for CNS gene therapy

由 Gray, Steven J, Woodard, Kenton T, Samulski, R Jude
發表在 Ther Deliv (2010)

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

由 Bailey, Rachel M., Rozenberg, Alejandra, Gray, Steven J.
發表在 Brain Res (2020)

Viral Vectors for Gene Delivery to the Central Nervous System

由 Lentz, Thomas B., Gray, Steven J., Samulski, R. Jude
出版 2011

Recent Gene Therapy Advancements for Neurological Diseases

由 Kalburgi, Sahana Nagabhushan, Khan, Nadia N., Gray, Steven J.
發表在 Discov Med (2013)

Clinical Applications Involving CNS Gene Transfer

由 Kantor, Boris, McCown, Thomas, Leone, Paola, Gray, Steven J.
發表在 Adv Genet (2014)

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

由 Banning, Antje, König, Jan F., Gray, Steven J., Tikkanen, Ritva
發表在 Int J Mol Sci (2017)

Identification of Small Molecule Compounds for Pharmacological Chaperone Therapy of Aspartylglucosaminuria

由 Banning, Antje, Gülec, Christina, Rouvinen, Juha, Gray, Steven J., Tikkanen, Ritva
發表在 Sci Rep (2016)

Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy

由 Bailey, Rachel M., Armao, Diane, Nagabhushan Kalburgi, Sahana, Gray, Steven J.
發表在 Mol Ther Methods Clin Dev (2018)

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...

由 Banning, Antje, Zakrzewicz, Anna, Chen, Xin, Gray, Steven J., Tikkanen, Ritva
發表在 Cells (2021)

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

由 Gray, Steven J., Gerhardt, Jeannine, Doerfler, Walter, Small, Lawrence E., Fanning, Ellen
出版 2007

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

由 Gray, Steven J., Liu, Guoqi, Altman, Amy L., Small, Lawrence E., Fanning, Ellen
出版 2006

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

由 Gray, Steven J., Kalburgi, Sahana Nagabhushan, McCown, Thomas J., Samulski, R. Jude
出版 2013

Methods for Gene Transfer to the Central Nervous System

由 Kantor, Boris, Bailey, Rachel M., Wimberly, Keon, Kalburgi, Sahana N., Gray, Steven J.
發表在 Adv Genet (2014)

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

由 Armao, Diane, Bailey, Rachel M., Bouldin, Thomas W., Kim, Yongbaek, Gray, Steven J.
發表在 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

由 Karumuthil-Melethil, Subha, Marshall, Michael S., Heindel, Clifford, Jakubauskas, Benas, Bongarzone, Ernesto, Gray, Steven J.
發表在 J Neurosci Res (2016)

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

由 Hewitt, F. Curtis, Li, Chengwen, Gray, Steven J., Cockrell, Shelley, Washburn, Michael, Samulski, R. Jude
出版 2009

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

由 Gray, Steven J, Matagne, Valerie, Bachaboina, Lavanya, Yadav, Swati, Ojeda, Sergio R, Samulski, R Jude
出版 2011

Optical coherence tomography features in brothers with aspartylglucosaminuria

由 Goodspeed, Kimberly, Harder, Lana, Hughes, Samuel, Conger, Darrel, Taravella, Julia, Gray, Steven J, Minassian, Berge
發表在 Ann Clin Transl Neurol (2018)

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

由 Chen, Xin, Snanoudj-Verber, Sarah, Pollard, Laura, Hu, Yuhui, Cathey, Sara S., Tikkanen, Ritva, Gray, Steven J.
發表在 Mol Ther (2021)