Autosomal dominant optic atrophy: A novel treatment for <i>OPA1</i> splice defects using U1 snRNA adaption.

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第一作者： Christoph,Jüschke

第一单位： Human Genetics, Faculty of Medicine and Health Sciences, University of Oldenburg, 26129 Oldenburg, Germany.

作者： Christoph,Jüschke ^[1] ; Thomas,Klopstock ^[2] ; Claudia B,Catarino ^[3] ; Marta,Owczarek-Lipska ^[4] ; Bernd,Wissinger ^[5] ; John,Neidhardt ^[6]

作者单位： Human Genetics, Faculty of Medicine and Health Sciences, University of Oldenburg, 26129 Oldenburg, Germany. ^[1] Friedrich-Baur Institute, Department of Neurology, University Hospital, LMU Munich, University of Munich, 80336 Munich, Germany.;German Center for Neurodegenerative Diseases (DZNE), 81377 Munich, Germany.;Munich Cluster for Systems Neurology (SyNergy), 81377 Munich, Germany. ^[2] Friedrich-Baur Institute, Department of Neurology, University Hospital, LMU Munich, University of Munich, 80336 Munich, Germany. ^[3] Human Genetics, Faculty of Medicine and Health Sciences, University of Oldenburg, 26129 Oldenburg, Germany.;Research Center Neurosensory Science, University of Oldenburg, 26129 Oldenburg, Germany. ^[4] Institute for Ophthalmic Research, Center for Ophthalmology, University of Tübingen, 72076 Tübingen, Germany. ^[5] Human Genetics, Faculty of Medicine and Health Sciences, University of Oldenburg, 26129 Oldenburg, Germany.;Research Center Neurosensory Science, University of Oldenburg, 26129 Oldenburg, Germany.;Joint Research Training Group of the Faculty of Medicine and Health Sciences, University of Oldenburg, 26129 Oldenburg, Germany and University Medical Center Groningen, 9700 RB Groningen, the Netherlands. ^[6]

关键词 ADOA Autosomal Dominant Optic Atrophy DOA Dominant Optic Atrophy ExSpeU1 OPA1 Optic Atrophy Type 1 U1 snRNA gene therapy splicing

DOI 10.1016/j.omtn.2021.10.019

PMID 34853716

发布时间 2021-12-03

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