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Quantifying and mitigating motor phenotypes induced by antisense oligonucleotides in the central nervous system.
Moazami MP, Rembetsy-Brown JM, Sarli SL, McEachern HR, Wang F, Ohara M, Wagh A, Kelly K, Krishnamurthy PM, Weiss A, Marosfoi M, King RM, Motwani M, Gray-Edwards H, Fitzgerald KA, Brown RH, Watts JK. Moazami MP, et al. Among authors: king rm. Mol Ther. 2024 Dec 4;32(12):4401-4417. doi: 10.1016/j.ymthe.2024.10.024. Epub 2024 Oct 28. Mol Ther. 2024. PMID: 39460376 Free PMC article.
Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.
Tran H, Moazami MP, Yang H, McKenna-Yasek D, Douthwright CL, Pinto C, Metterville J, Shin M, Sanil N, Dooley C, Puri A, Weiss A, Wightman N, Gray-Edwards H, Marosfoi M, King RM, Kenderdine T, Fabris D, Bowser R, Watts JK, Brown RH Jr. Tran H, et al. Among authors: king rm. Nat Med. 2022 Jan;28(1):117-124. doi: 10.1038/s41591-021-01557-6. Epub 2021 Dec 23. Nat Med. 2022. PMID: 34949835 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. Among authors: king rm. 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.
Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model.
Benatti HR, Prestigiacomo RD, Taghian T, Miller R, King R, Gounis MJ, Celik U, Bertrand S, Tuominen S, Bierfeldt L, Parsley E, Gallagher J, Hall EF, McElroy AW, Sena-Esteves M, Khvorova A, Aronin N, Gray-Edwards HL. Benatti HR, et al. Mol Ther Methods Clin Dev. 2023 Sep 26;31:101122. doi: 10.1016/j.omtm.2023.101122. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37920238 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. Among authors: king rm. 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.
Novel Distal Emboli Protection Technology: The EmboTrap.
Chueh JY, Marosfoi MG, Brooks OW, King RM, Puri AS, Gounis MJ. Chueh JY, et al. Among authors: king rm. Interv Neurol. 2017 Oct;6(3-4):268-276. doi: 10.1159/000480668. Epub 2017 Sep 16. Interv Neurol. 2017. PMID: 29118805 Free PMC article.
Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis.
Taghian T, Batista AR, Kamper S, Caldwell M, Lilley L, Li H, Rodriguez P, Mesa K, Zheng S, King RM, Gounis MJ, Todeasa S, Maguire A, Martin DR, Sena-Esteves M, Meade TJ, Gray-Edwards HL. Taghian T, et al. Among authors: king rm. Mol Ther Methods Clin Dev. 2021 Aug 26;23:128-134. doi: 10.1016/j.omtm.2021.08.003. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34703836 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. Among authors: king rm. JCI Insight. 2021 Dec 22;6(24):e152203. doi: 10.1172/jci.insight.152203. JCI Insight. 2021. PMID: 34935646 Free PMC article.
171 results