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A novel exon-specific U1snRNA therapeutic strategy to prevent retinal degeneration in familial dysautonomia
A novel exon-specific U1snRNA therapeutic strategy to prevent retinal degeneration in familial dysautonomia
2021
Anil Chekuri
Elisabetta Morini
Emily M. Logan
Aram J. Krauson
Monica Salani
Giulia Romano
Federico Riccardi
Franco Pagani
Luk H. Vandenberghe
Susan A. Slaugenhaupt
Keywords:
Familial dysautonomia
therapeutic strategy
Exon
Medicine
Bioinformatics
Retinal degeneration
Correction
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