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1. Full Text Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
by Maguire, Albert M, MD and High, Katherine A, Prof and Auricchio, Alberto, Prof and Wright, J Fraser, PhD and Pierce, Eric A, MD and Testa, Francesco, MD and Mingozzi, Federico, PhD and Bennicelli, Jeannette L, PhD and Ying, Gui-shuang, PhD and Rossi, Settimio, MD and Fulton, Ann, Prof and Marshall, Kathleen A, COT and Banfi, Sandro, MD and Chung, Daniel C, DO and Morgan, Jessica IW, PhD and Hauck, Bernd, PhD and Zelenaia, Olga, PhD and Zhu, Xiaosong, MD and Raffini, Leslie, MD and Coppieters, Frauke, PhD and De Baere, Elfride, PhD and Shindler, Kenneth S, MD and Volpe, Nicholas J, Prof and Surace, Enrico M, DVM and Acerra, Carmela, BA and Lyubarsky, Arkady, PhD and Redmond, T Michael, PhD and Stone, Edwin, Prof and Sun, Junwei, MBA and McDonnell, Jennifer Wellman, MS and Leroy, Bart P, Prof and Simonelli, Francesca, MD and Bennett, Jean, Prof
Lancet, The, ISSN 0140-6736, 2009, Volume 374, Issue 9701, pp. 1597 - 1605
Summary Background Gene therapy has the potential to reverse disease or prevent further deterioration of vision in patients with incurable inherited retinal... 
Internal Medicine | VISUAL CYCLE | ISOMEROHYDROLASE | MEDICINE, GENERAL & INTERNAL | VISION | SAFETY | ROD | KINETICS | ISOMERASE | MUTATIONS | RETINITIS-PIGMENTOSA | BLINDNESS | Dependovirus - genetics | Nystagmus, Physiologic | Age Factors | Humans | Male | Optic Atrophy, Hereditary, Leber - genetics | Genetic Vectors - therapeutic use | Dark Adaptation | Dose-Response Relationship, Drug | Young Adult | Adult | Female | Blindness - congenital | Eye Proteins - genetics | Optic Atrophy, Hereditary, Leber - diagnosis | Safety | Child | cis-trans-Isomerases | Electroretinography | Treatment Outcome | Optic Atrophy, Hereditary, Leber - therapy | Mutation - genetics | Visual Acuity | Blindness - genetics | Disease Progression | Genetic Vectors - genetics | Carrier Proteins - genetics | Injections | Adolescent | Genetic Therapy - methods | Usage | Care and treatment | Genetic aspects | Leber's congenital amaurosis | Gene therapy | Health aspects | Studies | Eye movements | Patients | Eyes & eyesight | Light | Blindness | Index Medicus | Abridged Index Medicus
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2. Full Text MiRNeye: A microRNA expression atlas of the mouse eye
by Karali, Marianthi and Peluso, Ivana and Gennarino, Vincenzo A and Bilio, Marchesa and Verde, Roberta and Lago, Giampiero and Dollé, Pascal and Banfi, Sandro
BMC Genomics, ISSN 1471-2164, 12/2010, Volume 11, Issue 1, pp. 715 - 715
Background: MicroRNAs (miRNAs) are key regulators of biological processes. To define miRNA function in the eye, it is essential to determine a high-resolution... 
MAMMALIAN EYE | STEM-CELLS | SMALL RNAS | EPITHELIUM | ADULT NEWT | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | GENETICS & HEREDITY | RETINA | MIRNAS | DICER | IDENTIFICATION | LENS | Retinal Pigment Epithelium - metabolism | Iris - metabolism | Databases, Genetic | MicroRNAs - metabolism | Gene Expression Profiling | Ciliary Body - metabolism | Organ Specificity - genetics | Iris - cytology | In Situ Hybridization | Time Factors | Gene Expression Regulation, Developmental | Cornea - cytology | Eye - metabolism | Ciliary Body - cytology | Mice, Inbred C57BL | Eye - cytology | Eye - growth & development | Lens, Crystalline - metabolism | Cornea - metabolism | Animals | Mice | MicroRNAs - genetics | Internet | Lens, Crystalline - cytology | Retinal Pigment Epithelium - cytology | Eye | MicroRNA | Physiological aspects | Genetic aspects | Research | Genetic regulation | Natural history | Design | Cluster analysis | Medical research | Ontology | Rodents | Life sciences | Hybridization | Experiments | Manuscripts | Data bases | Index Medicus | Cornea | Biochemistry, Molecular Biology | Genomics | Organ Specificity | Retinal Pigment Epithelium | Life Sciences | Lens, Crystalline | Iris | Ciliary Body | MicroRNAs
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3. Full Text miR-340 inhibits tumor cell proliferation and induces apoptosis by targeting multiple negative regulators of p27 in non-small cell lung cancer
by Fernandez, S and Risolino, M and Mandia, N and Talotta, F and Soini, Y and Incoronato, M and Condorelli, G and Banfi, S and Verde, P
Oncogene, ISSN 0950-9232, 08/2014, Volume 34, Issue 25, pp. 3240 - 3250
MicroRNAs (miRNAs) control cell cycle progression by targeting the transcripts encoding for cyclins, CDKs and CDK inhibitors, such as p27(KIP1) (p27).p27... 
BIOCHEMISTRY & MOLECULAR BIOLOGY | CELL BIOLOGY | MIR-222 | OVEREXPRESSION | MESSENGER-RNA | ONCOLOGY | MIRNA | GENETICS & HEREDITY | OSTEOSARCOMA | DEGRADATION | EXPRESSION | PROGRESSION | MICRORNA | CYCLE | Carcinoma, Non-Small-Cell Lung - pathology | Lung Neoplasms - genetics | Cell Cycle - genetics | RNA-Binding Proteins - genetics | Cell Proliferation - genetics | Adenocarcinoma - pathology | Adenocarcinoma of Lung | Carcinoma, Non-Small-Cell Lung - genetics | Humans | Apoptosis - genetics | Lung Neoplasms - pathology | Up-Regulation - genetics | Cell Movement - genetics | Cell Line, Tumor | Adenocarcinoma - genetics | MicroRNAs - genetics | Cyclin-Dependent Kinase Inhibitor p27 - genetics | Development and progression | Tumor suppressor genes | Genetic aspects | MicroRNA | Lung cancer, Non-small cell | Properties | Cellular biology | MicroRNAs | Lung cancer | Apoptosis | Index Medicus | Pumilio | miR-340 | p27 | NSCLC | SKP2
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4. Full Text A Gene Network Regulating Lysosomal Biogenesis and Function
by Marco Sardiello and Michela Palmieri and Alberto di Ronza and Diego Luis Medina and Marta Valenza and Vincenzo Alessandro Gennarino and Chiara Di Malta and Francesca Donaudy and Valerio Embrione and Roman S. Polishchuk and Sandro Banfi and Giancarlo Parenti and Elena Cattaneo and Andrea Ballabio
Science, ISSN 0036-8075, 7/2009, Volume 325, Issue 5939, pp. 473 - 477
Lysosomes are organelles central to degradation and recycling processes in animal cells. Whether lysosomal activity is coordinated to respond to cellular needs... 
Molecules | Messenger RNA | Neurodegenerative diseases | Microscopy | Genes | HeLa cells | Lysosomes | Reports | Gene expression regulation | Cellular immunity | Cells | CELLS | MICROPHTHALMIA TRANSCRIPTION FACTOR | AMINO | MULTIDISCIPLINARY SCIENCES | DISEASE | NEURODEGENERATION | MODEL | CATHEPSIN-D | EXPRESSION | BRAIN | BETA | Consensus Sequence | Promoter Regions, Genetic | Humans | Lysosomes - genetics | Cells, Cultured | Gene Regulatory Networks | Lysosomes - physiology | Transcription Factors - metabolism | Animals | Chromatin Immunoprecipitation | Sucrose - metabolism | Basic Helix-Loop-Helix Leucine Zipper Transcription Factors - metabolism | Mice | HeLa Cells | Inverted Repeat Sequences | Huntington's chorea | Development and progression | Research | DNA binding proteins | Properties | Gene expression | Enzymes | Cellular biology | Molecular biology | A gene | Index Medicus
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5. Full Text Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2
by Testa, Francesco, MD, PhD and Maguire, Albert M., MD and Rossi, Settimio, MD and Pierce, Eric A., MD, PhD and Melillo, Paolo, PhD and Marshall, Kathleen, COT and Banfi, Sandro, MD and Surace, Enrico M., DVM and Sun, Junwei, MS and Acerra, Carmela, BA and Wright, J. Fraser, PhD and Wellman, Jennifer, MS and High, Katherine A., MD and Auricchio, Alberto, MD and Bennett, Jean, MD, PhD and Simonelli, Francesca, MD
Ophthalmology, ISSN 0161-6420, 2013, Volume 120, Issue 6, pp. 1283 - 1291
Objective The aim of this study was to show the clinical data of long-term (3-year) follow-up of 5 patients affected by Leber congenital amaurosis type 2... 
Ophthalmology | VISUAL-ACUITY | ELECTRORETINOGRAPHY | SAFETY | FUNDUS AUTOFLUORESCENCE | DISEASE | OPHTHALMOLOGY | RPE65 MUTATIONS | GENE-THERAPY | STANDARD | Dependovirus - genetics | Genetic Therapy | Visual Fields - physiology | Follow-Up Studies | Leber Congenital Amaurosis - physiopathology | Tomography, Optical Coherence | Humans | Male | Leber Congenital Amaurosis - genetics | Reflex, Pupillary - physiology | Young Adult | Transfection | cis-trans-Isomerases - genetics | Adolescent | Adult | Female | Mutation | Genetic Vectors | Child | Leber Congenital Amaurosis - therapy | Visual Acuity - physiology | Injections, Intraocular | Medical colleges | Care and treatment | Genetic disorders | Blindness | Medical genetics | Index Medicus
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6. Full Text Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration
by Simonelli, Francesca and Maguire, Albert M and Testa, Francesco and Pierce, Eric A and Mingozzi, Federico and Bennicelli, Jeannette L and Rossi, Settimio and Marshall, Kathleen and Banfi, Sandro and Surace, Enrico M and Sun, Junwei and Redmond, T Michael and Zhu, Xiaosong and Shindler, Kenneth S and Ying, Gui-Shuang and Ziviello, Carmela and Acerra, Carmela and Wright, J Fraser and McDonnell, Jennifer Wellman and High, Katherine A and Bennett, Jean and Auricchio, Alberto
Molecular Therapy, ISSN 1525-0016, 03/2010, Volume 18, Issue 3, pp. 643 - 650
The safety and efficacy of gene therapy for inherited retinal diseases is being tested in humans affected with Leber's congenital amaurosis (LCA), an autosomal... 
MEDICINE, RESEARCH & EXPERIMENTAL | MOLECULAR-GENETICS | CANINE MODEL | VISION | RPE65 MUTATIONS | NYSTAGMUS | HEMOPHILIA | VARIABILITY | CHILDHOOD BLINDNESS | PHASE-I TRIAL | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | DISEASE | GENETICS & HEREDITY | Dependovirus - genetics | Retina - metabolism | Follow-Up Studies | Humans | Treatment Outcome | Leber Congenital Amaurosis - genetics | Disease Progression | Carrier Proteins - genetics | Time Factors | Vision, Ocular | Adult | Models, Genetic | Eye Proteins - genetics | Genetic Vectors | Transgenes | Leber Congenital Amaurosis - therapy | cis-trans-Isomerases | Genetic Therapy - methods | Index Medicus | Original
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7. Full Text miR-204 is required for lens and retinal development via Meis2 targeting
by Ivan Conte and Sabrina Carrella and Raffaella Avellino and Marianthi Karali and Raquel Macro-Ferreres and Paola Bovolenta and Sandro Banfi and Huda Y. Zoghbi
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 8/2010, Volume 107, Issue 35, pp. 15491 - 15496
MicroRNAs (miRNAs) are small noncoding RNAs that have important roles in the regulation of gene expression. The roles of individual miRNAs in controlling... 
Vertebrates | MicroRNA | Eyes | Messenger RNA | Developmental biology | Epithelial cells | Fiber cells | Optics | Retina | Embryos | Medaka fish | Eye development | MICRORNA FUNCTION | GENE | MULTIDISCIPLINARY SCIENCES | medaka fish | microRNA | eye development | CELL-FATE | PAX6 | PROLIFERATION | MOUSE RETINA | IDENTIFICATION | EXPRESSION | Cell Line | Retina - metabolism | Humans | Lens, Crystalline - embryology | Embryo, Nonmammalian - metabolism | Lens, Crystalline - metabolism | Embryo, Nonmammalian - embryology | Retina - embryology | Transcription Factors - genetics | Reverse Transcriptase Polymerase Chain Reaction | Homeodomain Proteins - genetics | Fish Proteins - genetics | Animals | In Situ Hybridization | Gene Expression Regulation, Developmental | Organogenesis - genetics | MicroRNAs - genetics | Models, Genetic | Oryzias - genetics | Oryzias - embryology | Genetic aspects | Research | Gene expression | Health aspects | Crystalline lens | Genotype & phenotype | Fish | Ribonucleic acid--RNA | Index Medicus | Biological Sciences
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8. Full Text A high-resolution anatomical atlas of the transcriptome in the mouse embryo
by Diez-Roux, Graciana and Banfi, Sandro and Sultan, Marc and Geffers, Lars and Anand, Santosh and Rozado, David and Magen, Alon and Canidio, Elena and Pagani, Massimiliano and Peluso, Ivana and Lin-Marq, Nathalie and Koch, Muriel and Bilio, Marchesa and Cantiello, Immacolata and Verde, Roberta and de Masi, Cristian and Bianchi, Salvatore A and Cicchini, Juliette and Perroud, Elodie and Mehmeti, Shprese and Dagand, Emilie and Schrinner, Sabine and Nürnberger, Asja and Schmidt, Katja and Metz, Katja and Zwingmann, Christina and Brieske, Norbert and Springer, Cindy and Hernandez, Ana Martinez and Herzog, Sarah and Grabbe, Frauke and Sieverding, Cornelia and Fischer, Barbara and Schrader, Kathrin and Brockmeyer, Maren and Dettmer, Sarah and Helbig, Christin and Alunni, Violaine and Battaini, Marie-Annick and Mura, Carole and Henrichsen, Charlotte N and Garcia-Lopez, Raquel and Echevarria, Diego and Puelles, Eduardo and Garcia-Calero, Elena and Kruse, Stefan and Uhr, Markus and Kauck, Christine and Feng, Guangjie and Milyaev, Nestor and Ong, Chuang Kee and Kumar, Lalit and Lam, MeiSze and Semple, Colin A and Gyenesei, Attila and Mundlos, Stefan and Radelof, Uwe and Lehrach, Hans and Sarmientos, Paolo and Reymond, Alexandre and Davidson, Duncan R and Dollé, Pascal and Antonarakis, Stylianos E and Yaspo, Marie-Laure and Martinez, Salvador and Baldock, Richard A and Eichele, Gregor and Ballabio, Andrea
PLoS Biology, ISSN 1544-9173, 2011, Volume 9, Issue 1, pp. e1000582 - e1000582
Ascertaining when and where genes are expressed is of crucial importance to understanding or predicting the physiological role of genes and proteins and how... 
NERVOUS-SYSTEM | FOREBRAIN | PROSOMERIC MODEL | DATABASE | BIOCHEMISTRY & MOLECULAR BIOLOGY | GENE-EXPRESSION PATTERNS | BIOLOGY | MECHANISMS | KIDNEY | WNT | VIEW | BRAIN | Mice - anatomy & histology | Mice - genetics | Animals | Atlases as Topic | Embryo, Mammalian | Mice, Inbred C57BL | Databases, Genetic | Gene Expression Profiling | Internet | Organ Specificity | Mice - embryology | Proteins | Brain research | Disease | Ontology | Rodents | Technological change | Genetics | Genomes | Gene expression | Index Medicus | Life Sciences | Mice | Development Biology
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9. Full Text Identification and Characterization of MicroRNAs Expressed in the Mouse Eye
by Karali, Marianthi and Peluso, Ivana and Marigo, Valeria and Banfi, Sandro
Investigative Ophthalmology & Visual Science, ISSN 0146-0404, 02/2007, Volume 48, Issue 2, pp. 509 - 515
PURPOSE. MicroRNAs ( miRNAs) are a class of small, endogenous RNAs that negatively regulate gene expression post-transcriptionally by binding to target sites... 
ATLAS | GENE-EXPRESSION | OPHTHALMOLOGY | MIRNAS | Retina - metabolism | Cornea - embryology | Lens, Crystalline - embryology | Lens, Crystalline - metabolism | In Situ Hybridization, Fluorescence | Retina - embryology | Cornea - metabolism | Animals | Retina - cytology | Cornea - cytology | Cell Differentiation | Mice | MicroRNAs - genetics | Oligonucleotide Probes - chemistry | Lens, Crystalline - cytology | Microscopy, Fluorescence | Databases, Factual | Gene Expression Regulation, Developmental - physiology | Index Medicus
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10. Full Text TGF-β controls MIR-181/ERK regulatory network during retinal axon specification and growth
by Carrella, Sabrina and Barbato, Sara and D'Agostino, Ylenia and Salierno, Francesco Giuseppe and Manfredi, Anna and Banfi, Sandro and Conte, Ivan
PLoS ONE, ISSN 1932-6203, 12/2015, Volume 10, Issue 12, pp. e0144129 - e0144129
Retinal axon specification and growth are critically sensitive to the dosage of numerous signaling molecules and transcription factors. Subtle variations in... 
PROGRAMMED CELL-DEATH | ORYZIAS-LATIPES | CONES | MULTIDISCIPLINARY SCIENCES | IN-VIVO | NEURONS | MECHANISMS | MEDAKA | EXPRESSION | PROMOTE | CHEMOTROPIC RESPONSES | MAP Kinase Signaling System - physiology | Animals | Axons - metabolism | Fish Proteins - metabolism | MicroRNAs - metabolism | rhoA GTP-Binding Protein - metabolism | Retina - embryology | Oryzias - embryology | Transforming Growth Factor beta - metabolism | Gene Expression Regulation, Developmental - physiology | Regulators | Transcription factors | Visual pathways | Retina | Kinases | Inactivation | Neurogenesis | Visual perception | Wiring | Visual system | Specifications | Genetics | Fish | Growth factors | Chromosomes | Deactivation | Developmental biology | MiRNA | Visual signals | Extracellular signal-regulated kinase | MAP kinase | RhoA protein | Gene expression | Medicine | Signaling | Functional morphology | Neural networks | Laboratory animals | Apoptosis | Index Medicus
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11. Full Text UTRdb and UTRsite (RELEASE 2010): A collection of sequences and regulatory motifs of the untranslated regions of eukaryotic mRNAs
by Grillo, Giorgio and Turi, Antonio and Licciulli, Flavio and Mignone, Flavio and Liuni, Sabino and Banfi, Sandro and Gennarino, Vincenzo Alessandro and Horner, David S and Pavesi, Giulio and Picardi, Ernesto and Pesole, Graziano
Nucleic Acids Research, ISSN 0305-1048, 10/2009, Volume 38, Issue 1, pp. D75 - D80
The 5' and 3' untranslated regions of eukaryotic mRNAs (UTRs) play crucial roles in the post-transcriptional regulation of gene expression through the... 
ELEMENTS | DATABASE | BIOCHEMISTRY & MOLECULAR BIOLOGY | GENOME | RESOURCE | User-Computer Interface | Computational Biology - methods | Information Storage and Retrieval - methods | Humans | Computational Biology - trends | Databases, Genetic | Genome, Plant | Algorithms | Animals | 5' Untranslated Regions | Databases, Protein | Protein Isoforms | Software | Internet | 3' Untranslated Regions | Databases, Nucleic Acid | Index Medicus
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12. Full Text Pax6 Regulates Gene Expression in the Vertebrate Lens through miR-204
by Shaham, Ohad and Gueta, Karen and Mor, Eyal and Oren-Giladi, Pazit and Grinberg, Dina and Xie, Qing and Cvekl, Ales and Shomron, Noam and Davis, Noa and Keydar-Prizant, Maya and Raviv, Shaul and Pasmanik-Chor, Metsada and Bell, Rachel E and Levy, Carmit and Avellino, Raffaella and Banfi, Sandro and Conte, Ivan and Ashery-Padan, Ruth
PLoS Genetics, ISSN 1553-7390, 2013, Volume 9, Issue 3, pp. e1003357 - e1003357
During development, tissue-specific transcription factors regulate both protein-coding and non-coding genes to control differentiation. Recent studies have... 
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