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Human Mutation
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November 9, 2018
Disease-causing variants of the conserved +2T of 5' splice sites can be rescued by engineered U1snRNAs
Daniela Scalet, Iva Maestri, Alessio Branchini, et al.
Molecular Therapy. Nucleic Acids
|
October 5, 2016
An Exon-Specific U1snRNA Induces a Robust Factor IX Activity in Mice Expressing Multiple Human FIX Splicing Mutants
Dario Balestra, Daniela Scalet, Franco Pagani, et al.
Human Molecular Genetics
|
June 12, 2015
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, et al.
Biochimica Et Biophysica Acta. Molecular Basis of Disease
|
September 19, 2016
Exploring Splicing-Switching Molecules For Seckel Syndrome Therapy
Daniela Scalet, Dario Balestra, Sara Rohban, et al.
Journal of Human Genetics
|
March 3, 2018
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, et al.
International Journal of Molecular Sciences
|
April 5, 2020
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, et al.
Page
of 1
Search research articles
Search
Showing results (1-10 of 6) with videos related to
Sort By:
Page
of 1
Human Mutation
|
November 9, 2018
Disease-causing variants of the conserved +2T of 5' splice sites can be rescued by engineered U1snRNAs
Daniela Scalet, Iva Maestri, Alessio Branchini, et al.
Molecular Therapy. Nucleic Acids
|
October 5, 2016
An Exon-Specific U1snRNA Induces a Robust Factor IX Activity in Mice Expressing Multiple Human FIX Splicing Mutants
Dario Balestra, Daniela Scalet, Franco Pagani, et al.
Human Molecular Genetics
|
June 12, 2015
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, et al.
Biochimica Et Biophysica Acta. Molecular Basis of Disease
|
September 19, 2016
Exploring Splicing-Switching Molecules For Seckel Syndrome Therapy
Daniela Scalet, Dario Balestra, Sara Rohban, et al.
Journal of Human Genetics
|
March 3, 2018
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, et al.
International Journal of Molecular Sciences
|
April 5, 2020
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, et al.
Page
of 1