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Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic Leukodystrophy Arsa Knock-Out Mice.
St Martin T, Seabrook TA, Gall K, Newman J, Avila N, Hayes A, Kivaa M, Lotterhand J, Mercaldi M, Patel K, Rivas IJ, Woodcock S, Wright TL, Seymour AB, Francone OL, Gingras J. St Martin T, et al. Among authors: seabrook ta. J Neurosci. 2023 May 10;43(19):3567-3581. doi: 10.1523/JNEUROSCI.1829-22.2023. Epub 2023 Mar 28. J Neurosci. 2023. PMID: 36977578 Free PMC article.
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial.
Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Servais L, Xiong H, Zanoteli E, Baranello G, Bruno C, Day JW, Deconinck N, Klein A, Mercuri E, Vlodavets D, Wang Y, Dodman A, El-Khairi M, Gorni K, Jaber B, Kletzl H, Gaki E, Fontoura P, Darras BT; FIREFISH Study Group. Masson R, et al. Lancet Neurol. 2022 Dec;21(12):1110-1119. doi: 10.1016/S1474-4422(22)00339-8. Epub 2022 Oct 14. Lancet Neurol. 2022. PMID: 36244364 Clinical Trial.
The National Conference on Health Disparities Student Research Forum.
Ford ME, Malek AM, Martino E, Abraham-Hilaire L, Ariyo O, Burshell D, Callwood G, Campbell L, Cannady K, Chavis C, Crawford B, Edwards A, Findlay V, Finley R, Greenaway C, Hazelton T, Hill M, Howard M, Knight KD, Lopez-Littleton V, Moore L, Randle D, Rivers DE, Salley JD, Seabrook T, Slaughter S, Stukes JB, Thorpe RJ Jr, Ragster L. Ford ME, et al. Among authors: seabrook t. J Cancer Educ. 2023 Feb;38(1):85-95. doi: 10.1007/s13187-021-02082-3. Epub 2021 Oct 16. J Cancer Educ. 2023. PMID: 34655025 Free PMC article.
Risdiplam in Type 1 Spinal Muscular Atrophy.
Baranello G, Darras BT, Day JW, Deconinck N, Klein A, Masson R, Mercuri E, Rose K, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Seabrook T, Fontoura P, Servais L; FIREFISH Working Group. Baranello G, et al. Among authors: seabrook t. N Engl J Med. 2021 Mar 11;384(10):915-923. doi: 10.1056/NEJMoa2009965. Epub 2021 Feb 24. N Engl J Med. 2021. PMID: 33626251 Clinical Trial.
Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study.
Annoussamy M, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Laugel V, Schara U, Gidaro T, Lilien C, Hogrel JY, Carlier P, Fournier E, Lowes L, Gorni K, Ly-Le Moal M, Hellbach N, Seabrook T, Czech C, Hermosilla R, Servais L; NatHis-SMA study group. Annoussamy M, et al. Among authors: seabrook t. Ann Clin Transl Neurol. 2021 Feb;8(2):359-373. doi: 10.1002/acn3.51281. Epub 2020 Dec 24. Ann Clin Transl Neurol. 2021. PMID: 33369268 Free PMC article.
Clade F AAVHSCs cross the blood brain barrier and transduce the central nervous system in addition to peripheral tissues following intravenous administration in nonhuman primates.
Ellsworth JL, Gingras J, Smith LJ, Rubin H, Seabrook TA, Patel K, Zapata N, Olivieri K, O'Callaghan M, Chlipala E, Morales P, Seymour A. Ellsworth JL, et al. Among authors: seabrook ta. PLoS One. 2019 Nov 26;14(11):e0225582. doi: 10.1371/journal.pone.0225582. eCollection 2019. PLoS One. 2019. PMID: 31770409 Free PMC article.
50 results