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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
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
Journal Article
Current Gene Therapy, ISSN 1566-5232, 07/2015, Volume 15, Issue 4, pp. 364 - 380
Human pluripotent stem cells represent a unique source for cell-based therapies and regenerative medicine. The intrinsic features of these cells such as their... 
Designer nucleases | Induced pluripotent stem (iPS) cells | Cell therapy | Regenerative medicine | Muscular dystrophies | Embryonic stem cells | Gene therapy | Muscle stem cells | PROGENITOR CELLS | embryonic stem cells | regenerative medicine | gene therapy | EMBRYONIC STEM | SATELLITE CELLS | muscle stem cells | designer nucleases | TEMPLATE DNA STRANDS | ALPHA-SARCOGLYCAN | SKELETAL-MUSCLE | MYOGENIC DIFFERENTIATION | GENETICS & HEREDITY | REGULATORY T-CELLS | DUCHENNE MUSCULAR-DYSTROPHY | muscular dystrophies | MYOBLAST TRANSPLANTATION | induced pluripotent stem (iPS) cells | Adult Stem Cells - physiology | Paired Box Transcription Factors - genetics | PAX3 Transcription Factor | Induced Pluripotent Stem Cells - physiology | Muscle, Skeletal - growth & development | MyoD Protein - genetics | Humans | Pluripotent Stem Cells - physiology | PAX7 Transcription Factor - genetics | Muscle, Skeletal - physiology | Muscle, Skeletal - cytology | Regeneration | Stem Cell Transplantation - methods | Animals | Muscular Diseases - physiopathology | Pluripotent Stem Cells - transplantation | Clustered Regularly Interspaced Short Palindromic Repeats | Cell Differentiation | Mice | Muscular Diseases - genetics | Adult Stem Cells - transplantation | Induced Pluripotent Stem Cells - cytology | Muscular Diseases - therapy | Genetic Therapy - methods
Journal Article
Journal Article
Archives of Disease in Childhood, ISSN 0022-2593, 01/2012, Volume 49, Issue 1, p. 27
Open lung biopsy is occasionally the only way to diagnose certain diffuse lung disorders in childhood. The clinical histories of 24 children with diffuse lung... 
Journal Article
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