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Targeted antisense oligonucleotide therapy rescues PRPF31 expression in retinitis pigmentosa caused by a splicing mutation.

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Title: Targeted antisense oligonucleotide therapy rescues PRPF31 expression in retinitis pigmentosa caused by a splicing mutation.
Authors: Banik P; Institute of Molecular Genetics, Czech Academy of Sciences, CZ 142 00 Prague, Czech Republic., Zimmann F; Institute of Molecular Genetics, Czech Academy of Sciences, CZ 142 00 Prague, Czech Republic., Thakur PK; Institute of Molecular Genetics, Czech Academy of Sciences, CZ 142 00 Prague, Czech Republic; Department of Ecology, Environment and Plant Sciences, Stockholm University, 106 91 Stockholm, Sweden., Dudakova L; Department of Pediatrics and Inherited Metabolic Disorders, First Faculty of Medicine, Charles University and General University Hospital in Prague, CZ 121 08 Prague, Czech Republic., Večerková K; Institute of Molecular Genetics, Czech Academy of Sciences, CZ 142 00 Prague, Czech Republic; Department of Informatics and Chemistry, University of Chemistry and Technology, CZ 166 28 Prague, Czech Republic., Kostov O; Department of Biochemistry, University of Colorado Boulder, Boulder, CO 80303, USA., Caruthers MH; Department of Biochemistry, University of Colorado Boulder, Boulder, CO 80303, USA., Kolář M; Institute of Molecular Genetics, Czech Academy of Sciences, CZ 142 00 Prague, Czech Republic; Department of Informatics and Chemistry, University of Chemistry and Technology, CZ 166 28 Prague, Czech Republic., Krejčířová I; Department of Pediatric Ophthalmology, University Hospital Brno and Faculty of Medicine, Masaryk University, CZ 625 00 Brno, Czech Republic., Vajter M; Department of Pediatrics and Inherited Metabolic Disorders, First Faculty of Medicine, Charles University and General University Hospital in Prague, CZ 121 08 Prague, Czech Republic; Department of Ophthalmology, First Faculty of Medicine, Charles University and General University Hospital in Prague, CZ 128 08 Prague, Czech Republic., Liskova P; Department of Pediatrics and Inherited Metabolic Disorders, First Faculty of Medicine, Charles University and General University Hospital in Prague, CZ 121 08 Prague, Czech Republic; Department of Ophthalmology, First Faculty of Medicine, Charles University and General University Hospital in Prague, CZ 128 08 Prague, Czech Republic., Bárta T; Department of Histology and Embryology, Faculty of Medicine, Masaryk University, CZ 625 00 Brno, Czech Republic., Staněk D; Institute of Molecular Genetics, Czech Academy of Sciences, CZ 142 00 Prague, Czech Republic. Electronic address: stanek@img.cas.cz.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2026 Jun 03. Date of Electronic Publication: 2026 Jun 03.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Targeted antisense oligonucleotide therapy rescues PRPF31 expression in retinitis pigmentosa caused by a splicing mutation.
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  Data: <searchLink fieldCode="AU" term="%22Banik+P%22">Banik P</searchLink>; Institute of Molecular Genetics, Czech Academy of Sciences, CZ 142 00 Prague, Czech Republic.<br /><searchLink fieldCode="AU" term="%22Zimmann+F%22">Zimmann F</searchLink>; Institute of Molecular Genetics, Czech Academy of Sciences, CZ 142 00 Prague, Czech Republic.<br /><searchLink fieldCode="AU" term="%22Thakur+PK%22">Thakur PK</searchLink>; Institute of Molecular Genetics, Czech Academy of Sciences, CZ 142 00 Prague, Czech Republic; Department of Ecology, Environment and Plant Sciences, Stockholm University, 106 91 Stockholm, Sweden.<br /><searchLink fieldCode="AU" term="%22Dudakova+L%22">Dudakova L</searchLink>; Department of Pediatrics and Inherited Metabolic Disorders, First Faculty of Medicine, Charles University and General University Hospital in Prague, CZ 121 08 Prague, Czech Republic.<br /><searchLink fieldCode="AU" term="%22Večerková+K%22">Večerková K</searchLink>; Institute of Molecular Genetics, Czech Academy of Sciences, CZ 142 00 Prague, Czech Republic; Department of Informatics and Chemistry, University of Chemistry and Technology, CZ 166 28 Prague, Czech Republic.<br /><searchLink fieldCode="AU" term="%22Kostov+O%22">Kostov O</searchLink>; Department of Biochemistry, University of Colorado Boulder, Boulder, CO 80303, USA.<br /><searchLink fieldCode="AU" term="%22Caruthers+MH%22">Caruthers MH</searchLink>; Department of Biochemistry, University of Colorado Boulder, Boulder, CO 80303, USA.<br /><searchLink fieldCode="AU" term="%22Kolář+M%22">Kolář M</searchLink>; Institute of Molecular Genetics, Czech Academy of Sciences, CZ 142 00 Prague, Czech Republic; Department of Informatics and Chemistry, University of Chemistry and Technology, CZ 166 28 Prague, Czech Republic.<br /><searchLink fieldCode="AU" term="%22Krejčířová+I%22">Krejčířová I</searchLink>; Department of Pediatric Ophthalmology, University Hospital Brno and Faculty of Medicine, Masaryk University, CZ 625 00 Brno, Czech Republic.<br /><searchLink fieldCode="AU" term="%22Vajter+M%22">Vajter M</searchLink>; Department of Pediatrics and Inherited Metabolic Disorders, First Faculty of Medicine, Charles University and General University Hospital in Prague, CZ 121 08 Prague, Czech Republic; Department of Ophthalmology, First Faculty of Medicine, Charles University and General University Hospital in Prague, CZ 128 08 Prague, Czech Republic.<br /><searchLink fieldCode="AU" term="%22Liskova+P%22">Liskova P</searchLink>; Department of Pediatrics and Inherited Metabolic Disorders, First Faculty of Medicine, Charles University and General University Hospital in Prague, CZ 121 08 Prague, Czech Republic; Department of Ophthalmology, First Faculty of Medicine, Charles University and General University Hospital in Prague, CZ 128 08 Prague, Czech Republic.<br /><searchLink fieldCode="AU" term="%22Bárta+T%22">Bárta T</searchLink>; Department of Histology and Embryology, Faculty of Medicine, Masaryk University, CZ 625 00 Brno, Czech Republic.<br /><searchLink fieldCode="AU" term="%22Staněk+D%22">Staněk D</searchLink>; Institute of Molecular Genetics, Czech Academy of Sciences, CZ 142 00 Prague, Czech Republic. Electronic address: stanek@img.cas.cz.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Cell+Press%22">Cell Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>100890581 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1525-0024 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2215250016%22">15250016 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther <i>Subsets: </i>MEDLINE
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        Value: 10.1016/j.ymthe.2026.05.033
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              Text: 2026 Jun 03
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