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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. Among authors: sena esteves m. 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.
AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates.
Hocquemiller M, Giersch L, Mei X, Gross AL, Randle AN, Gray-Edwards HL, Hudson JA, Todeasa S, Stoica L, Martin DR, Sena-Esteves M, Aiach K, Laufer R. Hocquemiller M, et al. Among authors: sena esteves m. Mol Ther Methods Clin Dev. 2022 Oct 7;27:281-292. doi: 10.1016/j.omtm.2022.10.004. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36320411 Free PMC article.
EZH2 inhibition reactivates epigenetically silenced FMR1 and normalizes molecular and electrophysiological abnormalities in fragile X syndrome neurons.
Fang M, Deibler SK, Krishnamurthy PM, Wang F, Rodriguez P, Banday S, Virbasius CM, Sena-Esteves M, Watts JK, Green MR. Fang M, et al. Among authors: sena esteves m. Front Neurosci. 2024 Feb 21;18:1348478. doi: 10.3389/fnins.2024.1348478. eCollection 2024. Front Neurosci. 2024. PMID: 38449737 Free PMC article.
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: sena esteves m. EBioMedicine. 2023 Jun;92:104627. doi: 10.1016/j.ebiom.2023.104627. Epub 2023 May 31. EBioMedicine. 2023. PMID: 37267847 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: sena esteves m. Ann Neurol. 2023 Nov;94(5):969-986. doi: 10.1002/ana.26756. Epub 2023 Aug 16. Ann Neurol. 2023. PMID: 37526361
Expression of ALS-PFN1 impairs vesicular degradation in iPSC-derived microglia.
Funes S, Jung J, Gadd DH, Mosqueda M, Zhong J, Shankaracharya, Unger M, Stallworth K, Cameron D, Rotunno MS, Dawes P, Fowler-Magaw M, Keagle PJ, McDonough JA, Boopathy S, Sena-Esteves M, Nickerson JA, Lutz C, Skarnes WC, Lim ET, Schafer DP, Massi F, Landers JE, Bosco DA. Funes S, et al. Among authors: sena esteves m. Nat Commun. 2024 Mar 20;15(1):2497. doi: 10.1038/s41467-024-46695-w. Nat Commun. 2024. PMID: 38509062 Free PMC article.
Expression of ALS-PFN1 impairs vesicular degradation in iPSC-derived microglia.
Funes S, Gadd DH, Mosqueda M, Zhong J, Jung J, Shankaracharya, Unger M, Cameron D, Dawes P, Keagle PJ, McDonough JA, Boopathy S, Sena-Esteves M, Lutz C, Skarnes WC, Lim ET, Schafer DP, Massi F, Landers JE, Bosco DA. Funes S, et al. Among authors: sena esteves m. bioRxiv [Preprint]. 2023 Jun 1:2023.06.01.541136. doi: 10.1101/2023.06.01.541136. bioRxiv. 2023. Update in: Nat Commun. 2024 Mar 20;15(1):2497. doi: 10.1038/s41467-024-46695-w. PMID: 37398081 Free PMC article. Updated. Preprint.
161 results