Mutation-adapted U1 snRNA corrects a splicing error of the dopa decarboxylase gene.

Abstract

Aromatic l-amino acid decarboxylase (AADC) deficiency is an inborn error of monoamine neurotransmitter synthesis, which results in dopamine, serotonin, epinephrine and norepinephrine deficiencies. The DDC gene founder mutation IVS6 + 4A > T is highly prevalent in Chinese patients with AADC deficiency. In this study, we designed several U1 snRNA vectors to… (More)
DOI: 10.1093/hmg/ddw323

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