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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
by Brigida, Immacolata and Brigida, Immacolata and Zoccolillo, Matteo and Zoccolillo, Matteo and Cicalese, Maria Pia and Cicalese, Maria Pia and Pfajfer, Laurène and Pfajfer, Laurène and Barzaghi, Federica and Scala, Serena and Scala, Serena and Oleaga-Quintas, Carmen and Álvarez-Álvarez, Jesus A and Álvarez-Álvarez, Jesus A and Sereni, Lucia and Sereni, Lucia and Giannelli, Stefania and Giannelli, Stefania and Sartirana, Claudia and Sartirana, Claudia and Dionisio, Francesca and Dionisio, Francesca and Pavesi, Luca and Benavides-Nieto, Marta and Benavides-Nieto, Marta and Basso-Ricci, Luca and Basso-Ricci, Luca and Capasso, Paola and Capasso, Paola and Mazzi, Benedetta and Mazzi, Benedetta and Rosain, Jeremie and Marcus, Nufar and Marcus, Nufar and Lee, Yu Nee and Lee, Yu Nee and Somech, Raz and Somech, Raz and Degano, Massimo and Degano, Massimo and Raiola, Giuseppe and Raiola, Giuseppe and Caorsi, Roberta and Picco, Paolo and Velez, Marcela Moncada and Moncada Velez, Marcela and Khourieh, Joelle and Arias, Andrés Augusto and Arias, Andrés Augusto and Bousfiha, Aziz and Bousfiha, Aziz and Issekutz, Thomas and Issekutz, Thomas and Issekutz, Andrew and Issekutz, Andrew and Boisson, Bertrand and Boisson, Bertrand and Dobbs, Kerry and Villa, Anna and Villa, Anna and Lombardo, Angelo and Lombardo, Angelo and Neven, Benedicte and Moshous, Despina and Moshous, Despina and Casanova, Jean-Laurent and Casanova, Jean-Laurent and Franco, José Luis and Franco, José Luis and Notarangelo, Luigi D and Scielzo, Cristina and Volpi, Stefano and Dupré, Loïc and Dupré, Loïc and Bustamante, Jacinta and Bustamante, Jacinta and Gattorno, Marco and Aiuti, Alessandro and Aiuti, Alessandro
Blood, ISSN 0006-4971, 11/2018, Volume 132, Issue 22, pp. 2362 - 2374
ARPC1B is a key factor for the assembly and maintenance of the ARP2/3 complex that is involved in actin branching from an existing filament. Germline biallelic... 
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
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Human Gene Therapy, ISSN 1043-0342, 11/2016, Volume 27, Issue 11, pp. A19 - A20
Journal Article
The Journal of clinical investigation, ISSN 0021-9738, 02/2019, Volume 130, Issue 4, pp. 1566 - 1580
BACKGROUND. The human bone marrow (BM) niche contains a population of mesenchymal stromal cells (MSCs) that provide physical support and regulate hematopoietic... 
Journal Article
Cytometry Part A, ISSN 1552-4922, 10/2017, Volume 91, Issue 10, pp. 952 - 965
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