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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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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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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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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