Publications by authors named "Daniela Scalet"

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A Compensatory U1snRNA Partially Rescues FAH Splicing and Protein Expression in a Splicing-Defective Mouse Model of Tyrosinemia Type I.
Dario Balestra, Daniela Scalet, Mattia Ferrarese, Silvia Lombardi, Nicole Ziliotto

Int J Mol Sci· March 2020

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Disease-causing variants of the conserved +2T of 5' splice sites can be rescued by engineered U1snRNAs.
Daniela Scalet, Iva Maestri, Alessio Branchini, Francesco Bernardi, Mirko Pinotti

Hum Mutat· January 2019

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The somatic FAH C.1061C>A change counteracts the frequent FAH c.1062+5G>A mutation and permits U1snRNA-based splicing correction.
Daniela Scalet, Claudia Sacchetto, Francesco Bernardi, Mirko Pinotti, Stan F J van de Graaf

J Hum Genet· May 2018

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Exploring Splicing-Switching Molecules For Seckel Syndrome Therapy.
Daniela Scalet, Dario Balestra, Sara Rohban, Matteo Bovolenta, Daniela Perrone

Biochim Biophys Acta Mol Basis Dis· January 2017

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An Exon-Specific U1snRNA Induces a Robust Factor IX Activity in Mice Expressing Multiple Human FIX Splicing Mutants.
Dario Balestra, Daniela Scalet, Franco Pagani, Malgorzata Ewa Rogalska, Rosella Mari

Mol Ther Nucleic Acids· October 2016

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Regulation of a strong F9 cryptic 5'ss by intrinsic elements and by combination of tailored U1snRNAs with antisense oligonucleotides.
Dario Balestra, Elena Barbon, Daniela Scalet, Nicola Cavallari, Daniela Perrone

Hum Mol Genet· September 2015

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