Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transduction. by Gabriella Cotugno, Patrizia Annunziata, Maria Vittoria Barone, Marianthi Karali, Sandro Banfi, Alberto Auricchio

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TGF-β Controls miR-181/ERK Regulatory Network during Retinal Axon Specification and Growth. by Sabrina Carrella, Sara Barbato, Ylenia D'Agostino, Francesco Giuseppe Salierno, Anna Manfredi, Sandro Banfi, Ivan Conte

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A Novel Variant in TUBB4B Causes Progressive Cone‐Rod Dystrophy and Early Onset Sensorineural Hearing Loss by Margherita Scarpato, Francesco Testa, Anna Nesti, Roberta Zeuli, Rosa Boccia, Gennaro Auletta, Sandro Banfi, Francesca Simonelli, Marianthi Karali

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miR-204 targeting of Ankrd13A controls both mesenchymal neural crest and lens cell migration. by Raffaella Avellino, Sabrina Carrella, Marinella Pirozzi, Maurizio Risolino, Francesco Giuseppe Salierno, Paola Franco, Patrizia Stoppelli, Pasquale Verde, Sandro Banfi, Ivan Conte

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In vitro high-content screening reveals miR-429 as a protective molecule in photoreceptor degeneration by Georgios Petrogiannakis, Irene Guadagnino, Santiago Negueruela, Martina Di Guida, Elena Marrocco, Mariateresa Pizzo, Annalaura Torella, Mariateresa Zanobio, Marianthi Karali, Diego Luis Medina, Sabrina Carrella, Sandro Banfi

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MicroRNA-restricted transgene expression in the retina. by Marianthi Karali, Anna Manfredi, Agostina Puppo, Elena Marrocco, Annagiusi Gargiulo, Mariacarmela Allocca, Michele Della Corte, Settimio Rossi, Massimo Giunti, Maria Laura Bacci, Francesca Simonelli, Enrico Maria Surace, Sandro Banfi, Alberto Auricchio

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Molecular diagnosis of Usher syndrome: application of two different next generation sequencing-based procedures. by Danilo Licastro, Margherita Mutarelli, Ivana Peluso, Kornelia Neveling, Nienke Wieskamp, Rossella Rispoli, Diego Vozzi, Emmanouil Athanasakis, Angela D'Eustacchio, Mariateresa Pizzo, Francesca D'Amico, Carmela Ziviello, Francesca Simonelli, Antonella Fabretto, Hans Scheffer, Paolo Gasparini, Sandro Banfi, Vincenzo Nigro

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Targeted long-read cDNA sequencing reveals novel splice-altering pathogenic variants causing retinal dystrophies by Dalila Capasso, Roberta Zeuli, Gavin Arno, Michael Kwint, Raoul Timmermans, Karla A. Ruiz-Ceja, Marianthi Karali, Francesca Simonelli, Sabrina Signorini, Enza Maria Valente, Frans P.M. Cremers, Sandro Banfi, Susanne Roosing, Daan M. Panneman, Suzanne E. de Bruijn

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Pax6 regulates gene expression in the vertebrate lens through miR-204. by Ohad Shaham, Karen Gueta, Eyal Mor, Pazit Oren-Giladi, Dina Grinberg, Qing Xie, Ales Cvekl, Noam Shomron, Noa Davis, Maya Keydar-Prizant, Shaul Raviv, Metsada Pasmanik-Chor, Rachel E Bell, Carmit Levy, Raffaella Avellino, Sandro Banfi, Ivan Conte, Ruth Ashery-Padan

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miR‐181a/b downregulation exerts a protective action on mitochondrial disease models by Alessia Indrieri, Sabrina Carrella, Alessia Romano, Alessandra Spaziano, Elena Marrocco, Erika Fernandez‐Vizarra, Sara Barbato, Mariateresa Pizzo, Yulia Ezhova, Francesca M Golia, Ludovica Ciampi, Roberta Tammaro, Jorge Henao‐Mejia, Adam Williams, Richard A Flavell, Elvira De Leonibus, Massimo Zeviani, Enrico M Surace, Sandro Banfi, Brunella Franco

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Union makes strength: a worldwide collaborative genetic and clinical study to provide a comprehensive survey of RD3 mutations and delineate the associated phenotype. by Isabelle Perrault, Alejandro Estrada-Cuzcano, Irma Lopez, Susanne Kohl, Shiqiang Li, Francesco Testa, Renate Zekveld-Vroon, Xia Wang, Esther Pomares, Jean Andorf, Nisrine Aboussair, Sandro Banfi, Nathalie Delphin, Anneke I den Hollander, Catherine Edelson, Ralph Florijn, Marc Jean-Pierre, Corinne Leowski, Andre Megarbane, Cristina Villanueva, Blanca Flores, Arnold Munnich, Huanan Ren, Ditta Zobor, Arthur Bergen, Rui Chen, Frans P M Cremers, Roser Gonzalez-Duarte, Robert K Koenekoop, Francesca Simonelli, Edwin Stone, Bernd Wissinger, Qingjiong Zhang, Josseline Kaplan, Jean-Michel Rozet

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A high-resolution anatomical atlas of the transcriptome in the mouse embryo. by Graciana Diez-Roux, Sandro Banfi, Marc Sultan, Lars Geffers, Santosh Anand, David Rozado, Alon Magen, Elena Canidio, Massimiliano Pagani, Ivana Peluso, Nathalie Lin-Marq, Muriel Koch, Marchesa Bilio, Immacolata Cantiello, Roberta Verde, Cristian De Masi, Salvatore A Bianchi, Juliette Cicchini, Elodie Perroud, Shprese Mehmeti, Emilie Dagand, Sabine Schrinner, Asja Nürnberger, Katja Schmidt, Katja Metz, Christina Zwingmann, Norbert Brieske, Cindy Springer, Ana Martinez Hernandez, Sarah Herzog, Frauke Grabbe, Cornelia Sieverding, Barbara Fischer, Kathrin Schrader, Maren Brockmeyer, Sarah Dettmer, Christin Helbig, Violaine Alunni, Marie-Annick Battaini, Carole Mura, Charlotte N Henrichsen, Raquel Garcia-Lopez, Diego Echevarria, Eduardo Puelles, Elena Garcia-Calero, Stefan Kruse, Markus Uhr, Christine Kauck, Guangjie Feng, Nestor Milyaev, Chuang Kee Ong, Lalit Kumar, MeiSze Lam, Colin A Semple, Attila Gyenesei, Stefan Mundlos, Uwe Radelof, Hans Lehrach, Paolo Sarmientos, Alexandre Reymond, Duncan R Davidson, Pascal Dollé, Stylianos E Antonarakis, Marie-Laure Yaspo, Salvador Martinez, Salvador Martinez, Richard A Baldock, Gregor Eichele, Andrea Ballabio

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