Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease.
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| Title: | Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease. |
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| Authors: | Stratton MP; Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL 60064, United States.; School of Graduate and Postdoctoral Studies, Rosalind Franklin University of Medicine and Science, North Chicago, IL 60064, United States., Centa JL; Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL 60064, United States.; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI 48109, United States., Swier VJ; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, United States., Pfeifer WL; Department of Ophthalmology and Visual Sciences, Institute for Vision Research, Carver College of Medicine, University of Iowa, IA City, IA 52242, United States., Booth CD; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, United States., Albert K; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, United States., Hunyara JL; Ionis Pharmaceuticals, Carlsbad, CA 92010, United States., Rechtzigel MJ; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, United States., Duelli FJ; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI 48109, United States., Leppert HG; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, United States., Rigo F; Ionis Pharmaceuticals, Carlsbad, CA 92010, United States., Smit T; Exemplar Genetics, Coralville, IA 52241, United States., Jafar-Nejad P; Ionis Pharmaceuticals, Carlsbad, CA 92010, United States., Weimer JM; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, United States.; Department of Pediatrics, Sanford School of Medicine, University of South Dakota, Sioux Falls, SD 57104, United States., Drack AV; Department of Ophthalmology and Visual Sciences, Institute for Vision Research, Carver College of Medicine, University of Iowa, IA City, IA 52242, United States.; Stead Family Department of Pediatrics, University of Iowa, IA City, IA 52242, United States., Hastings ML; Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL 60064, United States.; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI 48109, United States. |
| Source: | Nucleic acids research [Nucleic Acids Res] 2025 Oct 28; Vol. 53 (20). |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Oxford University Press Country of Publication: England NLM ID: 0411011 Publication Model: Print Cited Medium: Internet ISSN: 1362-4962 (Electronic) Linking ISSN: 03051048 NLM ISO Abbreviation: Nucleic Acids Res Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1362-4962 |
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| DOI: | 10.1093/nar/gkaf1141 |
