Autosomal dominant optic atrophy: A novel treatment for OPA1 splice defects using U1 snRNA adaption
Christoph Jüschke, Thomas Klopstock, Claudia B. Catarino, Marta Owczarek‐Lipska, Bernd Wissinger, John Neidhardt
Abstract
transcripts may suffice to overcome the haploinsufficiency.
Topics & Concepts
AtrophyspliceRNA splicingBiologyNeuroscienceSmall nuclear RNAGeneticsMedicineGeneRNARNA-dependent RNA polymeraseMitochondrial Function and PathologyRNA modifications and cancerRNA regulation and disease