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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.
Authors: Stratton MP; Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL, 60064, USA.; School of Graduate and Postdoctoral Studies, Rosalind Franklin University of Medicine and Science, North Chicago, IL, 60064, USA., Centa JL; Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL, 60064, USA.; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, 48109, USA., Swier VJ; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA., Pfeifer WL; Department of Ophthalmology and Visual Sciences, Institute for Vision Research, Carver College of Medicine, University of Iowa, Iowa City, IA, 52242, USA., Booth CD; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA., Albert K; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA., Hunyara JL; Ionis Pharmaceuticals, Carlsbad, CA, 92010, USA., Rechtzigel MJ; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA., Duelli FJ; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, 48109, USA., Leppert HG; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA., Rigo F; Ionis Pharmaceuticals, Carlsbad, CA, 92010, USA., Smit T; Exemplar Genetics, Coralville, IA 52241, USA., Jafar-Nejad P; Ionis Pharmaceuticals, Carlsbad, CA, 92010, USA., Weimer JM; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.; Department of Pediatrics, Sanford School of Medicine, University of South Dakota, Sioux Falls, SD, 57104, USA., Drack AV; Department of Ophthalmology and Visual Sciences, Institute for Vision Research, Carver College of Medicine, University of Iowa, Iowa City, IA, 52242, USA.; Stead Family Department of Pediatrics, University of Iowa, Iowa City, IA, 52242, USA., Hastings ML; Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL, 60064, USA.; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, 48109, USA.
Source: BioRxiv : the preprint server for biology [bioRxiv] 2025 May 31. Date of Electronic Publication: 2025 May 31.
Publication Type: Journal Article; Preprint
Journal Info: Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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PubType: Academic Journal
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  Data: Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease.
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  Data: <searchLink fieldCode="AU" term="%22Stratton+MP%22">Stratton MP</searchLink>; Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL, 60064, USA.; School of Graduate and Postdoctoral Studies, Rosalind Franklin University of Medicine and Science, North Chicago, IL, 60064, USA.<br /><searchLink fieldCode="AU" term="%22Centa+JL%22">Centa JL</searchLink>; Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL, 60064, USA.; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, 48109, USA.<br /><searchLink fieldCode="AU" term="%22Swier+VJ%22">Swier VJ</searchLink>; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.<br /><searchLink fieldCode="AU" term="%22Pfeifer+WL%22">Pfeifer WL</searchLink>; Department of Ophthalmology and Visual Sciences, Institute for Vision Research, Carver College of Medicine, University of Iowa, Iowa City, IA, 52242, USA.<br /><searchLink fieldCode="AU" term="%22Booth+CD%22">Booth CD</searchLink>; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.<br /><searchLink fieldCode="AU" term="%22Albert+K%22">Albert K</searchLink>; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.<br /><searchLink fieldCode="AU" term="%22Hunyara+JL%22">Hunyara JL</searchLink>; Ionis Pharmaceuticals, Carlsbad, CA, 92010, USA.<br /><searchLink fieldCode="AU" term="%22Rechtzigel+MJ%22">Rechtzigel MJ</searchLink>; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.<br /><searchLink fieldCode="AU" term="%22Duelli+FJ%22">Duelli FJ</searchLink>; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, 48109, USA.<br /><searchLink fieldCode="AU" term="%22Leppert+HG%22">Leppert HG</searchLink>; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.<br /><searchLink fieldCode="AU" term="%22Rigo+F%22">Rigo F</searchLink>; Ionis Pharmaceuticals, Carlsbad, CA, 92010, USA.<br /><searchLink fieldCode="AU" term="%22Smit+T%22">Smit T</searchLink>; Exemplar Genetics, Coralville, IA 52241, USA.<br /><searchLink fieldCode="AU" term="%22Jafar-Nejad+P%22">Jafar-Nejad P</searchLink>; Ionis Pharmaceuticals, Carlsbad, CA, 92010, USA.<br /><searchLink fieldCode="AU" term="%22Weimer+JM%22">Weimer JM</searchLink>; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, 57104, USA.; Department of Pediatrics, Sanford School of Medicine, University of South Dakota, Sioux Falls, SD, 57104, USA.<br /><searchLink fieldCode="AU" term="%22Drack+AV%22">Drack AV</searchLink>; Department of Ophthalmology and Visual Sciences, Institute for Vision Research, Carver College of Medicine, University of Iowa, Iowa City, IA, 52242, USA.; Stead Family Department of Pediatrics, University of Iowa, Iowa City, IA, 52242, USA.<br /><searchLink fieldCode="AU" term="%22Hastings+ML%22">Hastings ML</searchLink>; Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL, 60064, USA.; Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, 48109, USA.
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