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An Exon-Specific Small Nuclear U1 RNA (ExSpeU1) Improves Hepatic OTC Expression in a Splicing-Defective spf/ash Mouse Model of Ornithine Transcarbamylase Deficiency |
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| Authors: | Dario Balestra Mattia Ferrarese Silvia Lombardi Nicole Ziliotto Alessio Branchini Naomi Petersen Piter Bosma Mirko Pinotti Stan F J van de Graaf |
| Abstract: | |
| Keywords: | ornithine transcarbamylase deficiency OTCD splicing U1 mice AAV |