Steven J. Gray

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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