1. Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model. (25th April 2018) Authors: Donadon, Irving; Pinotti, Mirko; Rajkowska, Katarzyna; Pianigiani, Giulia; Barbon, Elena; Morini, Elisabetta; Motaln, Helena; Rogelj, Boris; Mingozzi, Federico; Slaugenhaupt, Susan A; Pagani, Franco Journal: Human molecular genetics Issue: Volume 27:Number 14(2018:Jul. 15) Page Start: 2466 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Exon‐Specific U1s Correct SPINK5 Exon 11 Skipping Caused by a Synonymous Substitution that Affects a Bifunctional Splicing Regulatory Element. Issue 5 (19th March 2015) Authors: Dal Mas, Andrea; Fortugno, Paola; Donadon, Irving; Levati, Lauretta; Castiglia, Daniele; Pagani, Franco Journal: Human mutation Issue: Volume 36:Issue 5(2015:May) Page Start: 504 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Rescue of spinal muscular atrophy mouse models with AAV9-Exon-specific U1 snRNA. Issue 14 (25th May 2019) Authors: Donadon, Irving; Bussani, Erica; Riccardi, Federico; Licastro, Danilo; Romano, Giulia; Pianigiani, Giulia; Pinotti, Mirko; Konstantinova, Pavlina; Evers, Melvin; Lin, Shuo; Rüegg, Markus A; Pagani, Franco Journal: Nucleic acids research Issue: Volume 47:Issue 14(2019) Page Start: 7618 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗