Suchergebnisse - "Pagani, Franco"

PubPharm (106)

1

Rescue of a familial dysautonomia mouse model by AAV9-Exon-specific U1 snRNA

enthalten in: American journal of human genetics | 2022

von Romano, G. | Riccardi, F. | Bussani, E. | +7

2

OTC intron 4 variations mediate pathogenic splicing patterns caused by the c.386G>A mutation in humans and spfash mice, and govern susceptibility to RNA-based therapies

enthalten in: Molecular medicine (Cambridge, Mass.) | 2021

von Sacchetto, C. | Peretto, L. | Baralle, F. | +7

3

OTC intron 4 variations mediate pathogenic splicing patterns caused by the c.386G>A mutation in humans and $ spf^{ash} $ mice, and govern susceptibility to RNA-based therapies

enthalten in: Molecular medicine | 2021

von Sacchetto, C. | Peretto, L. | Baralle, F. | +7

4

OTC intron 4 variations mediate pathogenic splicing patterns caused by the c.386G>A mutation in humans and $ spf^{ash} $ mice, and govern susceptibility to RNA-based therapies

enthalten in: Molecular medicine | 2021

von Sacchetto, C. | Peretto, L. | Baralle, F. | +7

5

OTC intron 4 variations mediate pathogenic splicing patterns caused by the c.386G>A mutation in humans and $ spf^{ash} $ mice, and govern susceptibility to RNA-based therapies

enthalten in: Molecular medicine | 2021

von Sacchetto, C. | Peretto, L. | Baralle, F. | +7

6

Rescue of common exon-skipping mutations in cystic fibrosis with modified U1 snRNAs

enthalten in: Human mutation | 2020

von Donegà, S. | Rogalska, M. | Pianigiani, G. | +3

7

A Compensatory U1snRNA Partially Rescues FAH Splicing and Protein Expression in a Splicing-Defective Mouse Model of Tyrosinemia Type I

enthalten in: International journal of molecular sciences | 2020

von Balestra, D. | Scalet, D. | Ferrarese, M. | +9

8

Rescue of common exon‐skipping mutations in cystic fibrosis with modified U1 snRNAs

enthalten in: Human mutation | 2020

von Donegà, S. | Rogalska, M. | Pianigiani, G. | +3

9

Rescue of spinal muscular atrophy mouse models with AAV9-Exon-specific U1 snRNA

enthalten in: Nucleic acids research | 2019

von Donadon, I. | Bussani, E. | Riccardi, F. | +9

10

Microprocessor-dependent processing of splice site overlapping microRNA exons does not result in changes in alternative splicing

enthalten in: RNA (New York, N.Y.) | 2018

von Pianigiani, G. | Licastro, D. | Fortugno, P. | +3

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