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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 Schmidt, Manfred and Verhoeyen, Els and Gobbo, Emilie and Divers, Dominique and Oudrhiri, Noufissa and Griscelli, Frank and Bennaceur-Griscelli, Annelise and Klatzmann, David and Anguela, Xavier M and Sharma, Rajiv and Sharma, Rajiv and Li, Hojun and Li, Hojun and Haurigot, Virginia and Haurigot, Virginia and Haurigot, Virginia and Bhagwat, Anand and Bhagwat, Anand and Davidson, Robert and Davidson, Robert and Zhou, Shangzhen and Zhou, Shangzhen and Zhou, Shangzhen and Doyon, Yannick and Gregory, Philip D and Gregory, Philip D and Gregory, Philip D and Holmes, Michael C and Holmes, Michael C and Holmes, Michael C and High, Katherine A and Carbonaro, Denise and Shaw, Kit and Jin, Xiangyang and Geiger, Sabine and Mishra, Suparna and Cooper, Aaron and DeOliveira, Satiro and Sokolic, Rob and Candotti, Fabio and Carmo, Marlene and Arumugam, Paritha and Alonso-Ferrero, Maria and Schambach, Axel and Schambach, Axel and Baum, Christopher and Baum, Christopher and Baum, Christopher and Risma, Kimberly and Malik, Punam and Jordan, Michael and Rivat, Christine and Booth, Claire and Thrasher, Adrian and Whilding, Lynsey and Archibald, Kyra and Oberg, Daniel and Golan, Talia and Hubert, Ayala and Shemi, Amotz and Khvalevsky, Elina Zorde and Gabai-Malka, Racheli and Focht, Gili and Brunschwig, Zivia and Raskin, Stephen and Goldberg, Nahum and Ben-David, Eli and Peretz, Tamar and Eliakim, Rami and Dankur, Alan and Galun and Rachmur, Itzik and Domb, Avi and Kopelman, Yael and Hantz, Yael and Lahav, Mor and Arbel-Alon, Sagit and Dickson, George and Barkats, Martine and Daboussi, Fayza and Silva, Georges and Cedrone, Frederic and Epinat, Jean Charles and Juillerat, Alexandre and Valton, Julien and Montini, Eugenio and Biffi, Alessandra and Biffi, Alessandra and Calabria, Andrea and Calabria, Andrea and Biasco, Luca and Biasco, Luca and Cesani, Martina and Cesani, Martina and Benedicenti, Fabrizio and Benedicenti, Fabrizio and Plati, Tiziana and Leo, Simone and Zanetti, Gianluigi and Aiuti, Alessandro and ...
Human Gene Therapy, ISSN 1043-0342, 10/2012, Volume 23, Issue 10, pp. A1 - A173
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 1/2011, Volume 108, Issue 4, pp. 1456 - 1461
Primary cilia are required for proper Sonic Hedgehog (Shh) signaling in mammals. However, their role in the signal transduction process remains unclear. We... 
Hedgehogs | Phenotypes | Neurons | Gene expression regulation | Physiological regulation | Genetic mutation | Embryos | Cells | Regulator genes | Cilia | Kinesin 2 | Cytoplasmic dynein 2 | Axoneme | Neural patterning | CELL BODY | MULTIDISCIPLINARY SCIENCES | MOUSE | NEURAL-TUBE | TRANSDUCTION | PRIMARY CILIUM | GLI3 | NEGATIVE REGULATOR | neural patterning | cytoplasmic dynein 2 | CAENORHABDITIS-ELEGANS | axoneme | KINESIN-II | IFT | kinesin 2 | Immunohistochemistry | Receptors, G-Protein-Coupled - metabolism | Zinc Finger Protein Gli2 | Zinc Finger Protein Gli3 | Hedgehog Proteins - metabolism | Male | Green Fluorescent Proteins - genetics | Intracellular Signaling Peptides and Proteins - metabolism | Embryo, Mammalian - metabolism | In Situ Hybridization | Hedgehog Proteins - genetics | Gene Expression Regulation, Developmental | Kruppel-Like Transcription Factors - metabolism | Mice, Mutant Strains | Smoothened Receptor | Cilia - ultrastructure | Female | Intracellular Signaling Peptides and Proteins - genetics | Fibroblasts - metabolism | Green Fluorescent Proteins - metabolism | Microscopy, Electron, Scanning | Signal Transduction | Mice, Inbred C57BL | Cells, Cultured | Nerve Tissue Proteins - genetics | Blotting, Western | Cilia - metabolism | Mice, Inbred C3H | Cilia - genetics | Nerve Tissue Proteins - metabolism | Proteins - genetics | Animals | Proteins - metabolism | Embryo, Mammalian - embryology | Embryo, Mammalian - cytology | Fibroblasts - cytology | Mice | Receptors, G-Protein-Coupled - genetics | Kruppel-Like Transcription Factors - genetics | Microscopy, Fluorescence | Physiological aspects | Cellular signal transduction | Genetic aspects | Research | Carrier proteins | Flagella (Microbiology) | Biological Sciences
Journal Article
Journal Article
Magnetic Resonance in Medicine, ISSN 0740-3194, 04/2014, Volume 71, Issue 4, pp. 1478 - 1488
Journal Article
SCIENTIFIC REPORTS, ISSN 2045-2322, 05/2019, Volume 9, Issue 1, pp. 7486 - 10
Certain point-mutations in the human SERPINA1-gene can cause severe alpha 1-antitrypsin-deficiency (A1AT-D). Affected individuals can suffer from... 
POLYMERS | LETHALITY | MECHANISM | ALPHA-ANTITRYPSIN DEFICIENCY | MULTIDISCIPLINARY SCIENCES | ALPHA-1-ANTITRYPSIN | MICE | POLYMERIZATION | MODEL | Animal models | Phenotypes | Liver diseases | Lung diseases | Environmental factors | Elastase | Mutation
Journal Article
Stem Cells International, ISSN 1687-966X, 2011, Volume 2011, p. 924782
Direct reprogramming of somatic cells into pluripotent cells by retrovirus-mediated expression of OCT4, SOX2, KLF4, and C-MYC is a promising approach to derive... 
DIRECTED DIFFERENTIATION | SOMATIC-CELLS | LIVER | MOUSE | GENERATION | HEPATOCYTE-LIKE CELLS | IMPRINTED GENES | FIBROBLASTS | TRANSPLANTATION | VECTOR | CELL & TISSUE ENGINEERING | Care and treatment | Liver diseases | Liver | Stem cells | Transplantation | Children | Research | Health aspects
Journal Article
MAGNETIC RESONANCE IN MEDICINE, ISSN 0740-3194, 04/2014, Volume 71, Issue 4, pp. 1478 - 1488
PurposeAt high magnetic field strengths (B-0 3 T), the shorter radiofrequency wavelength produces an inhomogeneous distribution of the transmit magnetic field.... 
TSE sequence | DESIGN | HUMAN BRAIN | EXCITATION PULSES | MRI | k(T)-points | 8 CHANNELS | T-2-weighted imaging | LOCAL SAR | B-1(+) inhomogeneity correction | B-1(+) INHOMOGENEITY | RF PULSES | 7 TESLA | PARALLEL-TRANSMISSION | RADIOLOGY, NUCLEAR MEDICINE & MEDICAL IMAGING
Journal Article
Fuel, ISSN 0016-2361, 02/2017, Volume 190, pp. 163 - 173
[Display omitted] •Experimental study of SCR spray/wall interaction under exhaust flow conditions.•The wall temperature and droplets kinetics experimentally... 
Infrared thermography | Phase Doppler anemometry | Spray/wall interaction | Urea-SCR | Heat transfer | Diesel deNOx | SCR | ENERGY & FUELS | UREA-WATER-SOLUTION | DECOMPOSITION | SPRAYS | ENGINEERING, CHEMICAL | EVAPORATION | INJECTION | PLATE
Journal Article
International Journal of Heat and Mass Transfer, ISSN 0017-9310, 05/2017, Volume 108, pp. 341 - 350
•Micro-CTs of real ceramic and metallic foams for exact determination of foam structural properties.•Micro-CTs of real foams for deriving equivalent Kelvin... 
Ceramic and metallic foams | Randomized Kelvin cell lattices | Mass transfer and flow simulation | CT-scans | Catalytic performance | HEAT-TRANSFER | PERFORMANCE | SUPPORTS | MODEL | SIMULATION | CERAMIC FOAMS | FLOW | ENGINEERING, MECHANICAL | PRESSURE-DROP | METAL FOAMS | PREDICTION | MECHANICS | THERMODYNAMICS
Journal Article
Nature Communications, ISSN 2041-1723, 08/2016, Volume 7, Issue 1, p. 12628
Human CtIP is a decisive factor in DNA double-strand break repair pathway choice by enabling DNA-end resection, the first step that differentiates homologous... 
E3 LIGASE | DAMAGE RESPONSE | STRAND BREAK REPAIR | NRF2 | STRUCTURAL BASIS | MULTIDISCIPLINARY SCIENCES | UBIQUITYLATION | KEAP1 | CELL-CYCLE | HOMOLOGOUS RECOMBINATION | INSIGHTS
Journal Article