Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model
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H. Motaln | F. Pagani | S. Slaugenhaupt | M. Pinotti | B. Rogelj | F. Mingozzi | E. Barbon | E. Morini | Irving Donadon | G. Pianigiani | K. Rajkowska | Elena Barbon