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Characterization and biodistribution of under-employed gene therapy vector AAV7.
Yost SA, Firlar E, Glenn JD, Carroll HB, Foltz S, Giles AR, Egley JM, Firnberg E, Cho S, Nguyen T, Henry WM, Janczura KJ, Bruder J, Liu Y, Danos O, Karumuthil-Melethil S, Pannem S, Yost V, Engelson Y, Kaelber JT, Dimant H, Smith JB, Mercer AC. Yost SA, et al. Among authors: danos o. J Virol. 2023 Nov 30;97(11):e0116323. doi: 10.1128/jvi.01163-23. Epub 2023 Oct 16. J Virol. 2023. PMID: 37843374 Free PMC article.
Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy.
Piechnik M, Amendum PC, Sawamoto K, Stapleton M, Khan S, Fnu N, Álvarez V, Pachon AMH, Danos O, Bruder JT, Karumuthil-Melethil S, Tomatsu S. Piechnik M, et al. Among authors: danos o. Int J Mol Sci. 2022 Oct 21;23(20):12693. doi: 10.3390/ijms232012693. Int J Mol Sci. 2022. PMID: 36293546 Free PMC article.
GluK2 Is a Target for Gene Therapy in Drug-Resistant Temporal Lobe Epilepsy.
Boileau C, Deforges S, Peret A, Scavarda D, Bartolomei F, Giles A, Partouche N, Gautron J, Viotti J, Janowitz H, Penchet G, Marchal C, Lagarde S, Trebuchon A, Villeneuve N, Rumi J, Marissal T, Khazipov R, Khalilov I, Martineau F, Maréchal M, Lepine A, Milh M, Figarella-Branger D, Dougy E, Tong S, Appay R, Baudouin S, Mercer A, Smith JB, Danos O, Porter R, Mulle C, Crépel V. Boileau C, et al. Among authors: danos o. Ann Neurol. 2023 Oct;94(4):745-761. doi: 10.1002/ana.26723. Epub 2023 Jul 6. Ann Neurol. 2023. PMID: 37341588
Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI.
Brunetti-Pierri N, Ferla R, Ginocchio VM, Rossi A, Fecarotta S, Romano R, Parenti G, Yildiz Y, Zancan S, Pecorella V, Dell'Anno M, Graziano M, Alliegro M, Andria G, Santamaria F, Brunetti-Pierri R, Simonelli F, Nigro V, Vargas M, Servillo G, Borgia F, Soscia E, Gargaro M, Funghini S, Tedesco N, Le Brun PR, Rupar CA, Prasad C, O'Callaghan M, Mitchell JJ, Danos O, Marteau JB, Galimberti S, Valsecchi MG, Veron P, Mingozzi F, Fallarino F, la Marca G, Sivri HS, Auricchio A. Brunetti-Pierri N, et al. Among authors: danos o. NEJM Evid. 2022 Jul;1(7):EVIDoa2200052. doi: 10.1056/EVIDoa2200052. Epub 2022 Jun 6. NEJM Evid. 2022. PMID: 38319253
Characterization of AAV-mediated dorsal root ganglionopathy.
Buss N, Lanigan L, Zeller J, Cissell D, Metea M, Adams E, Higgins M, Kim KH, Budzynski E, Yang L, Liu Y, Butt M, Danos O, Fiscella M. Buss N, et al. Among authors: danos o. Mol Ther Methods Clin Dev. 2022 Feb 1;24:342-354. doi: 10.1016/j.omtm.2022.01.013. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2022. PMID: 35229008 Free PMC article.
Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity.
Hooper AWM, Wong H, Niibori Y, Abdoli R, Karumuthil-Melethil S, Qiao C, Danos O, Bruder JT, Hampson DR. Hooper AWM, et al. Among authors: danos o. Mol Ther Methods Clin Dev. 2021 Jul 16;22:196-209. doi: 10.1016/j.omtm.2021.06.013. eCollection 2021 Sep 10. Mol Ther Methods Clin Dev. 2021. PMID: 34485605 Free PMC article.
212 results