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Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease.
Gray-Edwards HL, Randle AN, Maitland SA, Benatti HR, Hubbard SM, Canning PF, Vogel MB, Brunson BL, Hwang M, Ellis LE, Bradbury AM, Gentry AS, Taylor AR, Wooldridge AA, Wilhite DR, Winter RL, Whitlock BK, Johnson JA, Holland M, Salibi N, Beyers RJ, Sartin JL, Denney TS, Cox NR, Sena-Esteves M, Martin DR. Gray-Edwards HL, et al. Hum Gene Ther. 2018 Mar;29(3):312-326. doi: 10.1089/hum.2017.163. Epub 2017 Sep 18. Hum Gene Ther. 2018. PMID: 28922945
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.
Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, Gernoux G, Mueller C, Gray-Edwards HL, Moser RP, Bishop NC, Jaber SM, Gounis MJ, Sena-Esteves M, Pai AA, DiFiglia M, Aronin N, Khvorova A. Alterman JF, et al. Nat Biotechnol. 2019 Aug;37(8):884-894. doi: 10.1038/s41587-019-0205-0. Epub 2019 Aug 2. Nat Biotechnol. 2019. PMID: 31375812 Free PMC article.
A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna.
Taghian T, Marosfoi MG, Puri AS, Cataltepe OI, King RM, Diffie EB, Maguire AS, Martin DR, Fernau D, Batista AR, Kuchel T, Christou C, Perumal R, Chandra S, Gamlin PD, Bertrand SG, Flotte TR, McKenna-Yasek D, Tai PWL, Aronin N, Gounis MJ, Sena-Esteves M, Gray-Edwards HL. Taghian T, et al. Mol Ther. 2020 Feb 5;28(2):411-421. doi: 10.1016/j.ymthe.2019.11.012. Epub 2019 Nov 16. Mol Ther. 2020. PMID: 31813800 Free PMC article.
Canine Models of Inherited Musculoskeletal and Neurodegenerative Diseases.
Story BD, Miller ME, Bradbury AM, Million ED, Duan D, Taghian T, Faissler D, Fernau D, Beecy SJ, Gray-Edwards HL. Story BD, et al. Front Vet Sci. 2020 Mar 11;7:80. doi: 10.3389/fvets.2020.00080. eCollection 2020. Front Vet Sci. 2020. PMID: 32219101 Free PMC article. Review.
Natural history of Tay-Sachs disease in sheep.
Story B, Taghian T, Gallagher J, Koehler J, Taylor A, Randle A, Nielsen K, Gross A, Maguire A, Carl S, Johnson S, Fernau D, Diffie E, Cuddon P, Corado C, Chandra S, Sena-Esteves M, Kolodny E, Jiang X, Martin D, Gray-Edwards H. Story B, et al. Mol Genet Metab. 2021 Sep-Oct;134(1-2):164-174. doi: 10.1016/j.ymgme.2021.08.009. Epub 2021 Aug 21. Mol Genet Metab. 2021. PMID: 34456134 Free PMC article.
Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep.
Ferguson CM, Godinho BM, Alterman JF, Coles AH, Hassler M, Echeverria D, Gilbert JW, Knox EG, Caiazzi J, Haraszti RA, King RM, Taghian T, Puri A, Moser RP, Gounis MJ, Aronin N, Gray-Edwards H, Khvorova A. Ferguson CM, et al. JCI Insight. 2021 Dec 22;6(24):e152203. doi: 10.1172/jci.insight.152203. JCI Insight. 2021. PMID: 34935646 Free PMC article.
46 results