Steven J. Gray

Department of Pediatrics, UT Southwestern Medical Center

Affiliated withUT Southwestern Medical Center

Research Area

Biography

Steven J. Gray has not added Biography.
If you are Steven J. Gray and would like to personalize this page please email our Author Liaison for assistance.

JoVE Journal Publications

ArticleTotal : 1	Year
Publication title	2025

Other Publications

Article	Year
Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy. Molecular therapy. Methods & clinical development\| PubMed ID: 29766026	2018
Recent progress and considerations for AAV gene therapies targeting the central nervous system. Journal of neurodevelopmental disorders\| PubMed ID: 29776328	2018
Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model. Molecular therapy. Methods & clinical development\| PubMed ID: 30534578	2019
Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies. Brain research\| PubMed ID: 32289279	2020
Intellectual and Developmental Disabilities Research Centers: A Multidisciplinary Approach to Understand the Pathogenesis of Methyl-CpG Binding Protein 2-related Disorders. Neuroscience\| PubMed ID: 32360592	2020
Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 33186692	2021
Current Clinical Applications of In Vivo Gene Therapy with AAVs. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 33309881	2021
Giant axonal neuropathy: cross-sectional analysis of a large natural history cohort. Brain : a journal of neurology\| PubMed ID: 34114613	2021
Dual-isoform hUBE3A gene transfer improves behavioral and seizure outcomes in Angelman syndrome model mice. JCI insight\| PubMed ID: 34676830	2021
Long-term progression of retinal degeneration in a preclinical model of CLN7 Batten disease as a baseline for testing clinical therapeutics. EBioMedicine\| PubMed ID: 36374771	2022
Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies. The Journal of clinical investigation\| PubMed ID: 36951961	2023
Biochemical Correction of GM2 Ganglioside Accumulation in AB-Variant GM2 Gangliosidosis. International journal of molecular sciences\| PubMed ID: 37298170	2023
AAV-based in vivo gene therapy for neurological disorders. Nature reviews. Drug discovery\| PubMed ID: 37658167	2023
Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis. International journal of molecular sciences\| PubMed ID: 37834060	2023
RNAi Knockdown of in Maternal Expression of Prader-Willi Syndrome Genes. Genes\| PubMed ID: 39596566	2024
SURF1 Deficiency: Expanding on Disease Phenotype and Assessing Disease Burden by Describing Clinical and Biochemical Phenotype. American journal of medical genetics. Part A\| PubMed ID: 39632678	2025
Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency. Communications medicine\| PubMed ID: 39870870	2025