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FACS-based dual fluorescence reporter assay demonstrates efficacy of antisense oligonucleotide therapy of novel PRPF3 intronic splice variant.

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Title: FACS-based dual fluorescence reporter assay demonstrates efficacy of antisense oligonucleotide therapy of novel PRPF3 intronic splice variant.
Authors: Dolgin V; The Morris Kahn Laboratory of Human Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.; Genetics Institute, Soroka University Medical Center, Beer-Sheva, Israel.; The Shraga Segal Department of Microbiology, Immunology and Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel., Eremenko E; The Shraga Segal Department of Microbiology, Immunology and Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.; Zlotowski Neuroscience Center and Regenerative Medicine and Stem Cell Research Center, Ben-Gurion University of the Negev, Beer-Sheva, Israel., Narkis G; Genetics Institute, Soroka University Medical Center, Beer-Sheva, Israel.; The Shraga Segal Department of Microbiology, Immunology and Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel., Mazor-Oring M; The Morris Kahn Laboratory of Human Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.; Genetics Institute, Soroka University Medical Center, Beer-Sheva, Israel., Gradstein L; Department of Ophthalmology, Soroka Medical Center and Clalit Health Services, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel., Birk OS; The Morris Kahn Laboratory of Human Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.; Genetics Institute, Soroka University Medical Center, Beer-Sheva, Israel.; The Shraga Segal Department of Microbiology, Immunology and Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.; The Danek Gertner Institute of Human Genetics, Sheba Medical Center, Tel Hashomer, Israel.
Source: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2026 May 08; Vol. 37 (2), pp. 102949. Date of Electronic Publication: 2026 May 08 (Print Publication: 2026).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101581621 Publication Model: eCollection Cited Medium: Print ISSN: 2162-2531 (Print) Linking ISSN: 21622531 NLM ISO Abbreviation: Mol Ther Nucleic Acids Subsets: PubMed not MEDLINE
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  Data: FACS-based dual fluorescence reporter assay demonstrates efficacy of antisense oligonucleotide therapy of novel PRPF3 intronic splice variant.
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  Data: <searchLink fieldCode="AU" term="%22Dolgin+V%22">Dolgin V</searchLink>; The Morris Kahn Laboratory of Human Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.; Genetics Institute, Soroka University Medical Center, Beer-Sheva, Israel.; The Shraga Segal Department of Microbiology, Immunology and Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.<br /><searchLink fieldCode="AU" term="%22Eremenko+E%22">Eremenko E</searchLink>; The Shraga Segal Department of Microbiology, Immunology and Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.; Zlotowski Neuroscience Center and Regenerative Medicine and Stem Cell Research Center, Ben-Gurion University of the Negev, Beer-Sheva, Israel.<br /><searchLink fieldCode="AU" term="%22Narkis+G%22">Narkis G</searchLink>; Genetics Institute, Soroka University Medical Center, Beer-Sheva, Israel.; The Shraga Segal Department of Microbiology, Immunology and Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.<br /><searchLink fieldCode="AU" term="%22Mazor-Oring+M%22">Mazor-Oring M</searchLink>; The Morris Kahn Laboratory of Human Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.; Genetics Institute, Soroka University Medical Center, Beer-Sheva, Israel.<br /><searchLink fieldCode="AU" term="%22Gradstein+L%22">Gradstein L</searchLink>; Department of Ophthalmology, Soroka Medical Center and Clalit Health Services, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.<br /><searchLink fieldCode="AU" term="%22Birk+OS%22">Birk OS</searchLink>; The Morris Kahn Laboratory of Human Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.; Genetics Institute, Soroka University Medical Center, Beer-Sheva, Israel.; The Shraga Segal Department of Microbiology, Immunology and Genetics, Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel.; The Danek Gertner Institute of Human Genetics, Sheba Medical Center, Tel Hashomer, Israel.
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  Data: <searchLink fieldCode="JN" term="%22101581621%22">Molecular therapy. Nucleic acids</searchLink> [Mol Ther Nucleic Acids] 2026 May 08; Vol. 37 (2), pp. 102949. <i>Date of Electronic Publication: </i>2026 May 08 (<i>Print Publication: </i>2026).
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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>101581621 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Print <i>ISSN: </i>2162-2531 (Print) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2221622531%22">21622531 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther Nucleic Acids <i>Subsets: </i>PubMed not MEDLINE
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              Text: 2026 May 08
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