Antisense Oligonucleotide-Based Splicing Correction in Individuals with Leber Congenital Amaurosis due to Compound Heterozygosity for the c.2991+1655A>G Mutation in CEP290

Lonneke Duijkers, L Ingeborgh van den Born, John Neidhardt, Nathalie M Bax, Laurence H M Pierrache, B Jeroen Klevering, Rob W J Collin, Alejandro Garanto

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Medicine & Life Sciences