학술논문
'학술논문'
에서 검색결과 357건 | 목록
1~10
Academic Journal
Shieh, P.B.; Kuntz, N.L.; Dowling, J.J.; Müller-Felber, W.; Blaschek, A.; Bönnemann, C.G.; Foley, A.R.; Neuhaus, S.; Seferian, A.M.; Servais, L.; Smith, B.K.; Muntoni, F.; Saade, D.N.; Alfano, L.N.; Beggs, A.H.; Graham, R.J.; Buj-Bello, A.; Childers, M.K.; Duong, T.; Jain, M.; Coats, J.; James, E.S.; Lee, J.; Mavilio, F.; Miller, W.; Varfaj, F.; Murtagh, M.; Noursalehi, M.; Prasad, S.; Rico, S.; MacBean, V.; Han, C.; Lawlor, M.W.
In: The Lancet Neurology . (The Lancet Neurology, December 2023, 22(12):1125-1139)
Academic Journal
Andreoletti, G.; Chou, H.-J.; Sefid-Dashti, M.J.; Chen, C.; Lakshman, N.; Purushothaman, P.; Varfaj, F.; Urbinati, F.; Romano, O.; Grilli, A.; Mavilio, F.; Bicciato, S.
In: American Journal of Human Genetics . (American Journal of Human Genetics, 5 October 2023, 110(10):1648-1660)
Academic Journal
Brusson, M.; Chalumeau, A.; Martinucci, P.; Felix, T.; Ramadier, S.; Miccio, A.; Romano, O.; Mavilio, F.; Poletti, V.; Scaramuzza, S.; Ferrari, G.; Masson, C.; Cavazzana, M.; Amendola, M.
In: Molecular Therapy Nucleic Acids . (Molecular Therapy Nucleic Acids, 13 June 2023, 32:229-246)
Academic Journal
Mercado-Ayón, E.; Lynch, D.R.; Lin, H.; Warren, N.; Halawani, S.; Rodden, L.N.; Ngaba, L.; Dong, Y.N.; Chang, J.C.; Fonck, C.; Mavilio, F.
In: Frontiers in Neuroscience . (Frontiers in Neuroscience, 24 March 2022, 16)
Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicity
Academic Journal
Eggers, M.; Vannoy, C.H.; Huang, J.; Purushothaman, P.; Fonck, C.; Cunningham, J.; Sadhu, C.; Mavilio, F.; Brassard, J.; Meng, H.; Prom, M.J.; Lawlor, M.W.
In: EMBO Molecular Medicine . (EMBO Molecular Medicine, 11 January 2022, 14(1))
Academic Journal
Pavani, G.; Fabiano, A.; Laurent, M.; Amor, F.; Cantelli, E.; Tachtsidi, A.; Amendola, M.; Chalumeau, A.; Miccio, A.; Maule, G.; Cereseto, A.; Concordet, J.-P.; Mavilio, F.; Ferrari, G.
In: Blood Advances . (Blood Advances, 9 March 2021, 5(5):1137-1153)
Academic Journal
Weber, L.; Chalumeau, A.; Andre-Schmutz, I.; Cavazzana, M.; Frati, G.; Felix, T.; Hardouin, G.; Wollenschlaeger, C.; Meneghini, V.; Miccio, A.; Casini, A.; Cereseto, A.; Masson, C.; de Cian, A.; Concordet, J.-P.; Giovannangeli, C.; Mavilio, F.; Amendola, M.; El Nemer, W.
In: Science Advances . (Science Advances, 2020, 6(7))
Academic Journal
Charrier, S.; Poletti, V.; Cédrone, G.; Gjata, B.; Mavilio, F.; Galy, A.; Lagresle-Peyrou, C.; Cavazzana, M.; Hacein-Bey-Abina, S.; Rothe, M.; Schambach, A.; Fischer, A.; de Villartay, J.-P.
In: Molecular Therapy Methods and Clinical Development . (Molecular Therapy Methods and Clinical Development, 13 December 2019, 15:232-245)
Academic Journal
In Journal of Investigative Dermatology May 2018 138(5) Supplement:S137-S137
Academic Journal
Lattanzi, A.; Pavani, G.; Amor, F.; Amendola, M.; Miccio, A.; Meneghini, V.; Ramadier, S.; Felix, T.; Antoniani, C.; Mavilio, F.; Masson, C.; Alibeu, O.; Lee, C.; Bao, G.; Porteus, M.H.
In: Molecular Therapy . (Molecular Therapy, 2 January 2019, 27(1):137-150)
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[AR] MAVILIO, F.
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