Gray SJ - Search Results

1

Engineered microRNA-based regulatory element permits safe high-dose miniMECP2 gene therapy in Rett mice.

Sinnett SE, Boyle E, Lyons C, Gray SJ. Sinnett SE, et al. Among authors: gray sj. Brain. 2021 Nov 29;144(10):3005-3019. doi: 10.1093/brain/awab182. Brain. 2021. PMID: 33950254 Free PMC article.

2

Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery.

Sinnett SE, Hector RD, Gadalla KKE, Heindel C, Chen D, Zaric V, Bailey MES, Cobb SR, Gray SJ. Sinnett SE, et al. Among authors: gray sj. Mol Ther Methods Clin Dev. 2017 Apr 19;5:106-115. doi: 10.1016/j.omtm.2017.04.006. eCollection 2017 Jun 16. Mol Ther Methods Clin Dev. 2017. PMID: 28497072 Free PMC article.

3

Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome.

Gadalla KKE, Vudhironarit T, Hector RD, Sinnett S, Bahey NG, Bailey MES, Gray SJ, Cobb SR. Gadalla KKE, et al. Among authors: gray sj. Mol Ther Methods Clin Dev. 2017 Apr 22;5:180-190. doi: 10.1016/j.omtm.2017.04.007. eCollection 2017 Jun 16. Mol Ther Methods Clin Dev. 2017. PMID: 28497075 Free PMC article.

4

Recent endeavors in MECP2 gene transfer for gene therapy of Rett syndrome.

Sinnett SE, Gray SJ. Sinnett SE, et al. Among authors: gray sj. Discov Med. 2017 Oct;24(132):153-159. Discov Med. 2017. PMID: 29272692 Free article. Review.

5

Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies.

Bailey RM, Rozenberg A, Gray SJ. Bailey RM, et al. Among authors: gray sj. Brain Res. 2020 Jul 15;1739:146832. doi: 10.1016/j.brainres.2020.146832. Epub 2020 Apr 11. Brain Res. 2020. PMID: 32289279 Free PMC article. Review.

6

Intellectual and Developmental Disabilities Research Centers: A Multidisciplinary Approach to Understand the Pathogenesis of Methyl-CpG Binding Protein 2-related Disorders.

Fagiolini M, Patrizi A, LeBlanc J, Jin LW, Maezawa I, Sinnett S, Gray SJ, Molholm S, Foxe JJ, Johnston MV, Naidu S, Blue M, Hossain A, Kadam S, Zhao X, Chang Q, Zhou Z, Zoghbi H. Fagiolini M, et al. Among authors: gray sj. Neuroscience. 2020 Oct 1;445:190-206. doi: 10.1016/j.neuroscience.2020.04.037. Epub 2020 Apr 29. Neuroscience. 2020. PMID: 32360592 Free PMC article. Review.

7

The Efficacy of a Human-Ready miniMECP2 Gene Therapy in a Pre-Clinical Model of Rett Syndrome.

Sadhu C, Lyons C, Oh J, Jagadeeswaran I, Gray SJ, Sinnett SE. Sadhu C, et al. Among authors: gray sj. Genes (Basel). 2023 Dec 24;15(1):31. doi: 10.3390/genes15010031. Genes (Basel). 2023. PMID: 38254921 Free PMC article.

8

Recent progress and considerations for AAV gene therapies targeting the central nervous system.

Lykken EA, Shyng C, Edwards RJ, Rozenberg A, Gray SJ. Lykken EA, et al. Among authors: gray sj. J Neurodev Disord. 2018 May 18;10(1):16. doi: 10.1186/s11689-018-9234-0. J Neurodev Disord. 2018. PMID: 29776328 Free PMC article. Review.

9

Giant axonal neuropathy: cross-sectional analysis of a large natural history cohort.

Bharucha-Goebel DX, Norato G, Saade D, Paredes E, Biancavilla V, Donkervoort S, Kaur R, Lehky T, Fink M, Armao D, Gray SJ, Waite M, Debs S, Averion G, Hu Y, Zein WM, Foley AR, Jain M, Bönnemann CG. Bharucha-Goebel DX, et al. Among authors: gray sj. Brain. 2021 Nov 29;144(10):3239-3250. doi: 10.1093/brain/awab179. Brain. 2021. PMID: 34114613 Free PMC article.

10

Dual-isoform hUBE3A gene transfer improves behavioral and seizure outcomes in Angelman syndrome model mice.

Judson MC, Shyng C, Simon JM, Davis CR, Punt AM, Salmon MT, Miller NW, Ritola KD, Elgersma Y, Amaral DG, Gray SJ, Philpot BD. Judson MC, et al. Among authors: gray sj. JCI Insight. 2021 Oct 22;6(20):e144712. doi: 10.1172/jci.insight.144712. JCI Insight. 2021. PMID: 34676830 Free PMC article.

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