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by Visigalli, I and Cecere, F and Carriglio, N and Hernandez, R J and Hernandez, R J and De Simone, M and Vezzoli, M and Monti, L and Zino, E and Alfonso, E and Sanvito, F and Mauro, V and Norata, R and Acquati, S and Acquati, S and Redaelli, D and Redaelli, D and Dionisio, F and De Mattia, F and Scaramuzza, S and Rossi, C and Giannelli, S and Giannelli, S and Salerio, F and Sartirana, C and Brigida, I and Magni, S and Miglietta, S and Monteverde, S and Monti, I and Gregori, S and Ferrari, G and Aiuti, A and Castagnaro, L and Zancan, S and Cristofori, P and Naldini, L and Bonini, C and Waselle, L and Tissot, L and Sitjve, S and Büning, H and Ayuso, E and Déglon, N and Bueren, J and Trono, D and Nobrega, C and Mendonça, L and Marcelo, A and Lamazière, A and Tomé, S and Tomé, S and Déspres, G and Matos, C and Mechmet, F and Langui, D and den Dunnen, W and Pereira de Almeida, L and Cartier, N and Mitchell, J and Nemesh, J and Ghosh, S and Mello, C and McCaroll, S and Di Pasquale, G and Chandler, R J and Choi, E Y and Hubbard, B and Afione, S and Khalaj, M and Zheng, C and Grewe, B and Kaler, S G and Venditti, C P and Grimm, D and Grimm, D and Buj Bello, A and Nyns, E CA and Bingen, B O and Bart, C I and Kip, A and Poelma, R H and Volkers, L and Plomp, J J and Jangsangthong, W and Engels, M C and Schalij, M J and Zhang, G Q and Pijnappels, D A and Hildebrant, E and Penzes, J and Agbandje-McKenna, M and Gifford, R and Xiao, X and Akcakaya, P and Bobbin, M L and Guo, J A and Malagon-Lopez, J and Clement, K and Garcia, S P and ...
Human Gene Therapy, ISSN 1043-0342, 12/2018, Volume 29, Issue 12, pp. A1 - A169
Previously, results at 2 years after subretinal injection of a recombinant adeno-associated virus vector expressing RPE65 (rAAV2-CB-hRPE65) in eight adults and... 
Abstracts | MEDICINE, RESEARCH & EXPERIMENTAL | retinal degeneration | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | AAV | GENETICS & HEREDITY | gene therapy | Leber congenital amaurosis | RPE65
Journal Article
by Maffioletti, S M and Sarcar, S and Henderson, A and Mannhardt, I and Pinton, L and Moyle, L A and Moyle, L A and Steele-Stallard, H and Cappellari, O and Wells, K E and Ragazzi, M and Ragazzi, M and Ragazzi, M and Wang, W and Wang, W and Zammit, P and Wells, D J and Eschenhagen, T and Jung-Klawitter, S and Fuchs, N V and Upton, K R and Frömmrich, A and Miskey, C and Muñoz-Lopez, M and Shukla, R and Wang, J and Sebe, A and Merkert, S and Bock, A and Held, U and Menzel, M and Gogol-Döring, A and Haase, A and Izsvák, Z and Izsvák, Z and Ivics, Z and Martin, U and Garcia-Perez, J and Faulkner, G J and Terrasso, A P and Silva, M M and Arez, F and Sousa, M F and Raimundo, N and Gomes-Alves, P and Kremer, E J and Alves, P M and Brito, C and Moser, R and Decembrini, S and Gamm, D and Gamm, D and de Leone, V Ponce and Alves, S and Christaller, W and Subashi, E and Kacher, R and Lamazière, A and Despres, G and Saudou, F and Caboche, J and Betuing, S and Miyauchi, A and Nakajima, T and Taga, N and Ono, S A and Mizukami, H and Kato, M and Osaka, H and Muramatsu, S and Yamagata, T and Bouquet, C and Pruneau, D and Pruneau, D and Thomasson, N and Thomasson, N and Chavas, J and Chavas, J and Galluppi, F and Dalkara, D and Besnosman, R and Chenegros, G and Picaud, S and Sahel, J and Massó, A and Sánchez, A and Bosch, A and Blanch, R and Espinosa, J F and Giménez-Llort, L and Heidbüchel, J and Veinalde, R and Veinalde, R and Veinalde, R and Jäger, D and Jäger, D and Jäger, D and von Kalle, C and von Kalle, C and von Kalle, C and ...
Human Gene Therapy, ISSN 1043-0342, 12/2017, Volume 28, Issue 12, pp. A1 - A125
Journal Article
by Pañeda, A and Jiménez, I and Berlanga, A and Mork, A and Gaspar, B and Chuah, M K and Sgodda, M and Eggenschwiler, R and Möbus, S and Hoepfner, J and Kleinsorge, M and Papp, O and Sharma, A D and Broccoli, V and Broccoli, V and Tesar, P J and Dick, J E and De Luca, M and Birket, M and Meer, Bvan and Ribeiro, M and Passier, R and Bellin, M and Orlova, V and Davis, R and Freund, C and Kosmidis, G and Tertoolen, L and Giacca, M and Giacca, M and Goyenvalle, A and Leumann, C and Testa, G and Nehar, D and Courau, T and Qasim, W and Qasim, W and Sommermeyer, D and Liu, L and Maloney, D and Turtle, C and Maude, S L and Barrett, D M and Shaw, P A and Brogdon, J and Young, R and Marcucci, K T and Levine, B L and Frey, N and Porter, D L and Lacey, S F and Melenhorst, J J and Grupp, S A and Bonini, C and Friedmann-Morvinski, D and Soda, Y and Rommel, A and Myskiw, C and Kohn, D B and Wu, C and Espinoza, D A and Koelle, S J and Yang, D and Schlums, H and Kaur, A and Hammer, Q and Childs, R W and Romagnani, C and Bryceson, Y T and Scala, S and Scala, S and Scala, S and Pellin, D and Basso-Ricci, L and Basso-Ricci, L and Dionisio, F and Dionisio, F and Dionisio, F and Baricordi, C and Leonardelli, L and Leonardelli, L and Scaramuzza, S and Ferrua, F and Cicalese, M P and Giannelli, S and Giannelli, S and Neduva, V and Dow, D J and Roncarolo, M G and Ciceri, F and Ciceri, F and Vicard, P and Wit, E and Di Serio, C and Bonini, C and Naldini, L and Aiuti, A and Nelson, C E and Robinson-Hamm, J N and Bacchetta, Rosa and ...
Human Gene Therapy, ISSN 1043-0342, 11/2016, Volume 27, Issue 11, pp. A1 - A185
Journal Article
Journal Article
by Seymour, LW and Antoniou, M and Harbottle, Dr Richard P and Denning, C and Hajjar, R and Ferber, S and Searle, P F and Farzaneh, F and Krishnamurthy, P and Tye, G-J and Amofah, E and Barber, L and Chan, L and Mufti, GJ and Noble, A and Harrison, P and Falahati, Rustom and Zhang, Jianqing and Shi, Yimin and Flebbe-Rehwaldt, Linda and Gerson, Stanton L and Gaensler, Karin M L and Wilson, James M and von Kalle, C and Ivics, Z and Ammar, I and Voigt, K and Gogol-Döring, A and Miskey, C and Chen, W and Izsvák, Z and Cathomen, T and Cathomen, T and Jonuschies, J and Boldrin, L and Thrasher, A and Morgan, JE and Muntoni, F and Thrasher, A and Griesenbach, U and Weiss, D and Frances, K and Vassaux, G and Lombardo, A and Cesana, D and Cesana, D and Cesana, D and Cesana, D and Cesana, D and Genovese, P and Genovese, P and Genovese, P and Provasi, E and Colombo, DF and Neri, M and Magnani, Z and Cantore, C and Riso, P Lo and Damo, M and Holmes, MC and Holmes, MC and Gregory, PD and Gritti, A and Bonini, C and Naldini, L and Yáñez-Muñoz, RJ and Biffi, A and Sessa, M and Montini, E and Montini, E and Montini, E and Montini, E and Naldini, L and Wilson, James M and Baum, C and Modlich, U and Schambach, A and Galla, M and Maetzig, T and Kustikova, O and Brugman, M and Suerth, J and Heckl, D and Schwarzer, A and English, R and Wilson, J and Kernan, F and Meivar-Levy, I and Chernichovsky, E and Litichever, N and Ron, K and Sapir, T and Molakandov, K and Docherty, K and Lima, M João and Muir, K and Rashid, ST and Yusa, K and Strick-Marchand, H and Lomas, DA and ...
Human Gene Therapy, ISSN 1043-0342, 10/2011, Volume 22, Issue 10, pp. A1 - A138
Journal Article
by Sultan, S and Evans, M J and Antonitsis, P and Westaby, S and Anastasiadis, K and Gayral, M and Saland, E and Lulka, H and Vignolle-Vidoni, A and Hanoun, N and Lemarié, A and Ligat, L and Saint-Laurent, N and Lopez, F and Buscail, L and Sarry, J E and Casucci, M and Camisa, B and Falcone, L and Saudemont, A and Genovese, P and Naldini, L and Naldini, L and Ciceri, F and Bordignon, C and Bonini, C and Bonini, C and Bondanza, A and Cafferata, E A and Weber, H L and Werbajh, S and Salvatierra, E and Rotondaro, C and Sganga, L and Haab, G Acosta and Curiel, D T and Gidekel, M and Podhajcer, O L and Kaeppel, C and Wilkening, S and Rodriguez, M A Pañeda and Lopez-Franco, E and Fontanellas, A and Prieto, J and Spronck, L and von Kalle, C and Petry, H and Gonzalez-Aseguinolaza, G and Schmidt, M and Schreiber, C A and Sakuma, T and Izumiya, Y and Bressin, R K and Basu, U and Koide, K and Asokan, A and Fischer, S and Stiefel, S and Handrick, R and Hoerer, M and Otte, K and Kochanek, S and Halonen, P and Tarvainen, S and Tarvainen, S and Taavitsainen, J and Kuivanen, A and Koistinen, A and Laakkonen, J and Hartikainen, J and Yla-Herttuala, S and Enns, H and Jungmann, A and Leuchs, B and Volz, C and Schinkel, S and Koch, W J and Raake, P W and Most, P and Katus, H A and Müller, O J and Basilico, C and Jaulin, N and Leroux, I and Schleder, C and Zuber, C and Charrier, M and Lieubeau, B and Péréon, Y and Magot, A and Hamel, A and Adjali, O and Rouger, K and Genovese, P and Schiroli, G and Conway, A and Guschin, D and Escobar, G and Di Tomaso, T and Marin, V and ...
Human Gene Therapy, ISSN 1043-0342, 10/2015, Volume 26, Issue 10, pp. A-1 - A-108
Journal Article
Critical Reviews in Biochemistry and Molecular Biology, ISSN 1040-9238, 07/2017, Volume 52, Issue 4, pp. 355 - 380
Journal Article
Journal Article