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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.
Life-Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy.
Johnson AK, McCurdy VJ, Gray-Edwards HL, Maguire AS, Cochran JN, Gross AL, Skinner HE, Randle AN, Shirley JL, Brunson BL, Bradbury AM, Leroy SG, Hwang M, Rockwell HE, Cox NR, Baker HJ, Seyfried TN, Sena-Esteves M, Martin DR. Johnson AK, et al. Among authors: gray edwards hl. Ann Neurol. 2023 Nov;94(5):969-986. doi: 10.1002/ana.26756. Epub 2023 Aug 16. Ann Neurol. 2023. PMID: 37526361
A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis.
Kell P, Sidhu R, Qian M, Mishra S, Nicoli ER, D'Souza P, Tifft CJ, Gross AL, Gray-Edwards HL, Martin DR, Sena-Esteves M, Dietzen DJ, Singh M, Luo J, Schaffer JE, Ory DS, Jiang X. Kell P, et al. Among authors: gray edwards hl. EBioMedicine. 2023 Jun;92:104627. doi: 10.1016/j.ebiom.2023.104627. Epub 2023 May 31. EBioMedicine. 2023. PMID: 37267847 Free PMC article.
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.
46 results