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. |
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| 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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