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

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Bibliographic Details
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
Database: MEDLINE Ultimate
Description
ISSN:2162-2531
DOI:10.1016/j.omtn.2026.102949