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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 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
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 Alam, Shadab and Albareti, Franco D and Prieto, Carlos Allende and Anders, F and Anderson, Scott F and Anderton, Timothy and Andrews, Brett H and Armengaud, Eric and Aubourg, Éric and Bailey, Stephen and Basu, Sarbani and Bautista, Julian E and Beaton, Rachael L and Beers, Timothy C and Bender, Chad F and Berlind, Andreas A and Beutler, Florian and Bhardwaj, Vaishali and Bird, Jonathan C and Bizyaev, Dmitry and Blake, Cullen H and Blanton, Michael R and Blomqvist, Michael and Bochanski, John J and Bolton, Adam S and Bovy, Jo and Bradley, A. Shelden and Brandt, W.N and Brauer, D.E and Brinkmann, J and Brown, Peter J and Brownstein, Joel R and Burden, Angela and Burtin, Etienne and Busca, Nicolás G and Cai, Zheng and Capozzi, Diego and Rosell, Aurelio Carnero and Carr, Michael A and Carrera, Ricardo and Chambers, K.C and Chaplin, William James and Chen, Yen-Chi and Chiappini, Cristina and Chojnowski, S. Drew and Chuang, Chia-Hsun and Clerc, Nicolas and Comparat, Johan and Covey, Kevin and Croft, Rupert A. C and Cuesta, Antonio J and Cunha, Katia and Costa, Luiz N. Da and Rio, Nicola Da and Davenport, James R. A and Dawson, Kyle S and Lee, Nathan De and Delubac, Timothée and Deshpande, Rohit and Dhital, Saurav and Dutra-Ferreira, Letícia and Dwelly, Tom and Ealet, Anne and Ebelke, Garrett L and Edmondson, Edward M and Eisenstein, Daniel J and Ellsworth, Tristan and Elsworth, Yvonne and Epstein, Courtney R and Eracleous, Michael and Escoffier, Stephanie and Esposito, Massimiliano and Evans, Michael L and Fan, Xiaohui and Fernández-Alvar, Emma and Feuillet, Diane and Ak, Nurten Filiz and Finley, Hayley and Finoguenov, Alexis and Flaherty, Kevin and Fleming, Scott W and Font-Ribera, Andreu and Foster, Jonathan and Frinchaboy, Peter M and Galbraith-Frew, J.G and García, Rafael A and García-Hernández, D.A and Pérez, Ana E. García and Gaulme, Patrick and Ge, Jian and Génova-Santos, R and Georgakakis, A and Ghezzi, Luan and Gillespie, Bruce A and Girardi, Léo and Goddard, Daniel and Gontcho, Satya Gontcho A and Hernández, Jonay I. González and Grebel, Eva K and Green, Paul J and ...
Astrophysical Journal, Supplement Series, ISSN 0067-0049, 07/2015, Volume 219, Issue 1, pp. 12 - 27
Journal Article
by Ivezić, Željko and Kahn, Steven M and Tyson, J. Anthony and Abel, Bob and Acosta, Emily and Allsman, Robyn and Alonso, David and Alsayyad, Yusra and Anderson, Scott F and Andrew, John and Angel, James Roger P and Angeli, George Z and Ansari, Reza and Antilogus, Pierre and Araujo, Constanza and Armstrong, Robert and Arndt, Kirk T and Astier, Pierre and Aubourg, Eric and Auza, Nicole and Axelrod, Tim S and Bard, Deborah J and Barr, Jeff D and Barrau, Aurelian and Bartlett, James G and Bauer, Amanda E and Bauman, Brian J and Baumont, Sylvain and Bechtol, Ellen and Bechtol, Keith and Becker, Andrew C and Becla, Jacek and Beldica, Cristina and Bellavia, Steve and Bianco, Federica B and Biswas, Rahul and Blanc, Guillaume and Blazek, Jonathan and Blandford, Roger D and Bloom, Josh S and Bogart, Joanne and Bond, Tim W and Booth, Michael T and Borgland, Anders W and Borne, Kirk and Bosch, James F and Boutigny, Dominique and Brackett, Craig A and Bradshaw, Andrew and Brandt, William Nielsen and Brown, Michael E and Bullock, James S and Burchat, Patricia and Burke, David L and Cagnoli, Gianpietro and Calabrese, Daniel and Callahan, Shawn and Callen, Alice L and Carlin, Jeffrey L and Carlson, Erin L and Chandrasekharan, Srinivasan and Charles-Emerson, Glenaver and Chesley, Steve and Cheu, Elliott C and Chiang, Hsin-Fang and Chiang, James and Chirino, Carol and Chow, Derek and Ciardi, David R and Claver, Charles F and Cohen-Tanugi, Johann and Cockrum, Joseph J and Coles, Rebecca and Connolly, Andrew J and Cook, Kem H and Cooray, Asantha and Covey, Kevin R and Cribbs, Chris and Cui, Wei and Cutri, Roc and Daly, Philip N and Daniel, Scott F and Daruich, Felipe and Daubard, Guillaume and Daues, Greg and Dawson, William and Delgado, Francisco and Dellapenna, Alfred and Peyster, Robert De and Val-Borro, Miguel De and Digel, Seth W and Doherty, Peter and Dubois, Richard and Dubois-Felsmann, Gregory P and Durech, Josef and Economou, Frossie and Eifler, Tim and Eracleous, Michael and Emmons, Benjamin L and Neto, Angelo Fausti and ... and SLAC National Accelerator Lab., Menlo Park, CA (United States) and Brookhaven National Lab. (BNL), Upton, NY (United States) and Fysikum and Naturvetenskapliga fakulteten and Stockholms universitet and Oskar Klein-centrum för kosmopartikelfysik (OKC)
Astrophysical Journal, ISSN 0004-637X, 03/2019, Volume 873, Issue 2, p. 111
Journal Article
by Gott III, J. Richard and Mack III, Claude E and Ahn, Christopher P and Alexandroff, Rachael and Allende Prieto, Carlos and Anders, Friedrich and Anderson, Scott F and Anderton, Timothy and Andrews, Brett H and Aubourg, Éric and Bailey, Stephen and Bastien, Fabienne A and Bautista, Julian E and Beers, Timothy C and Beifiori, Alessandra and Bender, Chad F and Berlind, Andreas A and Beutler, Florian and Bhardwaj, Vaishali and Bird, Jonathan C and Bizyaev, Dmitry and Blake, Cullen H and Blanton, Michael R and Blomqvist, Michael and Bochanski, John J and Bolton, Adam S and Borde, Arnaud and Bovy, Jo and Bradley, Alaina Shelden and Brandt, W.N and Brauer, Dorothée and Brinkmann, J and Brownstein, Joel R and Busca, Nicolás G and Carithers, William and Carlberg, Joleen K and Carnero, Aurelio R and Carr, Michael A and Chiappini, Cristina and Chojnowski, S. Drew and Chuang, Chia-Hsun and Comparat, Johan and Crepp, Justin R and Cristiani, Stefano and Croft, Rupert A. C and Cuesta, Antonio J and Cunha, Katia and Da Costa, Luiz N and Dawson, Kyle S and De Lee, Nathan and Dean, Janice D. R and Delubac, Timothée and Deshpande, Rohit and Dhital, Saurav and Ealet, Anne and Ebelke, Garrett L and Edmondson, Edward M and Eisenstein, Daniel J and Epstein, Courtney R and Escoffier, Stephanie and Esposito, Massimiliano and Evans, Michael L and Fabbian, D and Fan, Xiaohui and Favole, Ginevra and Femenía Castellá, Bruno and Fernández Alvar, Emma and Feuillet, Diane and Filiz Ak, Nurten and Finley, Hayley and Fleming, Scott W and Font-Ribera, Andreu and Frinchaboy, Peter M and Galbraith-Frew, J.G and García-Hernández, D.A and Pérez, Ana E. García and Ge, Jian and Génova-Santos, R and Gillespie, Bruce A and Girardi, Léo and González Hernández, Jonay I and Gunn, James E and Guo, Hong and Halverson, Samuel and Harding, Paul and Harris, David W and Hasselquist, Sten and Hawley, Suzanne L and Hayden, Michael and Hearty, Frederick R and Herrero Davó, Artemio and Ho, Shirley and Hogg, David W and Holtzman, Jon A and Honscheid, Klaus and Huehnerhoff, Joseph and Ivans, Inese I and Jackson, Kelly M and Jiang, Peng and Johnson, Jennifer A and ...
Astrophysical Journal, Supplement Series, ISSN 0067-0049, 2014, Volume 211, Issue 2, pp. 17 - 16
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 Ahn, Christopher P and Alexandroff, Rachael and Allende Prieto, Carlos and Anderson, Scott F and Anderton, Timothy and Andrews, Brett H and Aubourg, Éric and Bailey, Stephen and Balbinot, Eduardo and Barnes, Rory and Bautista, Julian and Beers, Timothy C and Beifiori, Alessandra and Berlind, Andreas A and Bhardwaj, Vaishali and Bizyaev, Dmitry and Blake, Cullen H and Blanton, Michael R and Blomqvist, Michael and Bochanski, John J and Bolton, Adam S and Borde, Arnaud and Bovy, Jo and Brandt, W.N and Brinkmann, J and Brown, Peter J and Brownstein, Joel R and Bundy, Kevin and Busca, N.G and Carithers, William and Carnero, Aurelio R and Carr, Michael A and Casetti-Dinescu, Dana I and Chen, Yanmei and Chiappini, Cristina and Comparat, Johan and Connolly, Natalia and Crepp, Justin R and Cristiani, Stefano and Croft, Rupert A. C and Cuesta, Antonio J and Da Costa, Luiz N and Davenport, James R. A and Dawson, Kyle S and De Putter, Roland and De Lee, Nathan and Delubac, Timothée and Dhital, Saurav and Ealet, Anne and Ebelke, Garrett L and Edmondson, Edward M and Eisenstein, Daniel J and Escoffier, S and Esposito, Massimiliano and Evans, Michael L and Fan, Xiaohui and Femenía Castellá, Bruno and Fernández Alvar, Emma and Ferreira, Leticia D and Filiz Ak, N and Finley, Hayley and Fleming, Scott W and Font-Ribera, Andreu and Frinchaboy, Peter M and García-Hernández, D.A and Pérez, A. E. García and Ge, Jian and Génova-Santos, R and Gillespie, Bruce A and Girardi, Léo and González Hernández, Jonay I and Grebel, Eva K and Gunn, James E and Guo, Hong and Haggard, Daryl and Hamilton, Jean-Christophe and Harris, David W and Hawley, Suzanne L and Hearty, Frederick R and Ho, Shirley and Hogg, David W and Holtzman, Jon A and Honscheid, Klaus and Huehnerhoff, J and Ivans, Inese I and Ivezić, Željko and Jacobson, Heather R and Jiang, Linhua and Johansson, Jonas and Johnson, Jennifer A and Kauffmann, Guinevere and Kirkby, David and Kirkpatrick, Jessica A and Klaene, Mark A and Knapp, Gillian R and Kneib, Jean-Paul and Le Goff, Jean-Marc and Leauthaud, Alexie and Lee, Khee-Gan and Lee, Young Sun and ... and Fermi National Accelerator Lab. (FNAL), Batavia, IL (United States) and Lawrence Berkeley National Lab. (LBNL), Berkeley, CA (United States) and Brookhaven National Laboratory (BNL), Upton, NY (United States)
Astrophysical Journal, Supplement Series, ISSN 0067-0049, 12/2012, Volume 203, Issue 2, pp. 21 - 13
Journal Article