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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
Journal Article
Nature Cell Biology, ISSN 1465-7392, 09/2012, Volume 14, Issue 9, pp. 911 - 923
Mutations of the cyclin-dependent kinase-like 5 (CDKL5) and netrin-G1 (NTNG1) genes cause a severe neurodevelopmental disorder with clinical features that are... 
INFANTILE SPASMS | PROTEIN | RETT-SYNDROME | MENTAL-RETARDATION | GENE | PHOSPHORYLATION | ADHESION MOLECULES | MUTATIONS | EPILEPTIC ENCEPHALOPATHY | INTERSTITIAL DELETION | CELL BIOLOGY | Disks Large Homolog 4 Protein | Phosphorylation | Humans | Spine - metabolism | Cercopithecus aethiops | Molecular Sequence Data | Dendritic Spines - metabolism | Intracellular Signaling Peptides and Proteins - metabolism | Synapses - genetics | Excitatory Postsynaptic Potentials - physiology | Synapses - metabolism | HEK293 Cells | Neurons - physiology | Female | Membrane Proteins - metabolism | Neurons - metabolism | Spine - pathology | Intracellular Signaling Peptides and Proteins - genetics | Excitatory Postsynaptic Potentials - genetics | Protein-Serine-Threonine Kinases - metabolism | Amino Acid Sequence | Cell Adhesion - genetics | Membrane Proteins - genetics | Cells, Cultured | Protein-Serine-Threonine Kinases - genetics | Receptors, Cell Surface - metabolism | Nerve Tissue Proteins - genetics | Nerve Tissue Proteins - metabolism | Rett Syndrome - metabolism | Animals | Cell Adhesion - physiology | Dendritic Spines - pathology | Rett Syndrome - pathology | Glutamic Acid - metabolism | Mice | Mutation | Rett Syndrome - genetics | COS Cells | Receptors, Cell Surface - genetics | Gene mutations | Physiological aspects | Genetic aspects | Research | Health aspects | Protein kinases | Synapses
Journal Article
Cell Reports, ISSN 2211-1247, 02/2018, Volume 22, Issue 8, pp. 2066 - 2079
Journal Article
Cell Stem Cell, ISSN 1934-5909, 07/2018, Volume 23, Issue 1, pp. 13 - 14
In a recent issue of , perform a systematic screening to identify several transcription factor pairs able to generate a variety of different induced neuronal... 
REGULATORY NETWORKS | DIRECT CONVERSION | HUMAN FIBROBLASTS | GENERATION | DOPAMINERGIC-NEURONS | CELL & TISSUE ENGINEERING | CELL BIOLOGY
Journal Article
Journal of the Neurological Sciences, ISSN 0022-510X, 2015, Volume 357, pp. e43 - e43
Journal Article
Neurobiology of Disease, ISSN 0969-9961, 2015, Volume 81, pp. 144 - 153
Abstract Pantothenate kinase-associated neurodegeneration is an early onset autosomal recessive movement disorder caused by mutation of the pantothenate... 
Neurology | NBIA | Induced neurons | Iron metabolism | Mitochondria | Reactive oxygen species | PANK2 | NERVOUS-SYSTEM | HALLERVORDEN-SPATZ DISEASE | SULFUR | PANK2 MUTATIONS | NEUROSCIENCES | DEFICIENCY | PERMEABILITY TRANSITION | CLUSTER BIOGENESIS | MOUSE MODEL | ACCUMULATION | PARKINSONS-DISEASE | Aconitate Hydratase - metabolism | Neurons - pathology | Reactive Oxygen Species - metabolism | Glutathione - metabolism | Humans | Fibroblasts - ultrastructure | Mitochondria - ultrastructure | Lip - metabolism | Neurons - ultrastructure | Adenosine Triphosphate - metabolism | Membrane Potential, Mitochondrial - physiology | Adult | Energy Metabolism - physiology | Infant, Newborn | Neurodegenerative Diseases - pathology | Oxidation-Reduction | Cells, Cultured | Neurodegenerative Diseases - genetics | Phosphotransferases (Alcohol Group Acceptor) - genetics | Mitochondria - metabolism | Mitochondria - pathology | Fibroblasts - pathology | Iron - metabolism | Analysis of Variance | Mutation | Neurodegenerative Diseases - enzymology | Enzymes | Neurons | Analysis | PKAN, pantothenate kinase associated neurodegeneration | TMRM, tetramethylrhodamine methyl ester | FAC, ferric ammonium citrate | iNs, induced neurons | LIP, labile iron pool | RPA, rhodamine B-[(1,10-phenanthroline-5-yl)-aminocarbonyl]benzyl ester | DCF, dichlorofluorescein | DHR-123, dihydrorhodamine 123 | IRP1, iron regulatory protein 1 | RPAC, rhodamine B-[(phenanthren-9-yl)-aminocarbonyl]-benzylester | PIH, pyridoxal isonicotinoyl hydrazone | DFO, deferoxamine | NBIA, neurodegeneration with brain iron accumulation | ROS, reactive oxygen species
Journal Article
SCIENCE TRANSLATIONAL MEDICINE, ISSN 1946-6234, 06/2012, Volume 4, Issue 140
Mesoangioblasts are stem/progenitor cells derived from a subset of pericytes found in muscle that express alkaline phosphatase. They have been shown to... 
PLURIPOTENT STEM-CELLS | MEDICINE, RESEARCH & EXPERIMENTAL | SKELETAL-MUSCLE | THERAPY | MULTIPOTENT | REGENERATION | MESOANGIOBLASTS | GENERATION | GENE-TRANSFER | PERICYTES | ALPHA-SARCOGLYCAN | CELL BIOLOGY
Journal Article
Cell, ISSN 0092-8674, 09/2017, Volume 171, Issue 1, pp. 229 - 241.e15
Journal Article
Journal Article