Rescue of a familial dysautonomia mouse model by AAV9-Exon-specific U1 snRNA
Giulia Romano, Federico Riccardi, Erica Bussani, Simone Vodret, Danilo Licastro, Isabella Ragone, Giuseppe Ronzitti, Elisabetta Morini, Susan Slaugenhaupt, Franco Pagani
Topics & Concepts
ExonBiologyDysautonomiaFamilial dysautonomiaRNA splicingAlternative splicingMessenger RNAAdeno-associated virusRNAMolecular biologyGeneCell biologyGeneticsInternal medicineMedicineDiseaseRecombinant DNAVector (molecular biology)Hereditary Neurological DisordersRNA regulation and diseaseGenetic Neurodegenerative Diseases