Department of Pediatrics,
Department of Neurology,
Department of Molecular Biology,
McDermott Center for Human Growth and Development
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Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy.
Molecular therapy. Methods & clinical development Jun, 2018 | Pubmed ID: 29766026
Recent progress and considerations for AAV gene therapies targeting the central nervous system.
Journal of neurodevelopmental disorders May, 2018 | Pubmed ID: 29776328
Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model.
Molecular therapy. Methods & clinical development Mar, 2019 | Pubmed ID: 30534578
Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies.
Brain research Jul, 2020 | Pubmed ID: 32289279
Intellectual and Developmental Disabilities Research Centers: A Multidisciplinary Approach to Understand the Pathogenesis of Methyl-CpG Binding Protein 2-related Disorders.
Neuroscience Oct, 2020 | Pubmed ID: 32360592
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 Mar, 2021 | Pubmed ID: 33186692
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Molecular therapy : the journal of the American Society of Gene Therapy Feb, 2021 | Pubmed ID: 33309881
Giant axonal neuropathy: cross-sectional analysis of a large natural history cohort.
Brain : a journal of neurology Nov, 2021 | Pubmed ID: 34114613
Dual-isoform hUBE3A gene transfer improves behavioral and seizure outcomes in Angelman syndrome model mice.
JCI insight Oct, 2021 | Pubmed ID: 34676830
Long-term progression of retinal degeneration in a preclinical model of CLN7 Batten disease as a baseline for testing clinical therapeutics.
EBioMedicine Nov, 2022 | Pubmed ID: 36374771
Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies.
The Journal of clinical investigation May, 2023 | Pubmed ID: 36951961
Biochemical Correction of GM2 Ganglioside Accumulation in AB-Variant GM2 Gangliosidosis.
International journal of molecular sciences May, 2023 | Pubmed ID: 37298170
AAV-based in vivo gene therapy for neurological disorders.
Nature reviews. Drug discovery Oct, 2023 | Pubmed ID: 37658167
Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis.
International journal of molecular sciences Sep, 2023 | Pubmed ID: 37834060
RNAi Knockdown of in Maternal Expression of Prader-Willi Syndrome Genes.
Genes Oct, 2024 | Pubmed ID: 39596566
SURF1 Deficiency: Expanding on Disease Phenotype and Assessing Disease Burden by Describing Clinical and Biochemical Phenotype.
American journal of medical genetics. Part A Apr, 2025 | Pubmed ID: 39632678
Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency.
Communications medicine Jan, 2025 | Pubmed ID: 39870870
UT Southwestern Medical Center
Nanda L. Regmi1,
Xin Chen1,
Rachel M. Bailey1,2,
Steven J. Gray1,3,4,5
1Department of Pediatrics, UT Southwestern Medical Center,
2Center for Alzheimer's and Neurodegenerative Diseases, UT Southwestern Medical Center,
3Department of Neurology, UT Southwestern Medical Center,
4Department of Molecular Biology, UT Southwestern Medical Center,
5McDermott Center for Human Growth and Development, UT Southwestern Medical Center
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