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AAV-Delivered RNAi Targeting Mutant LDB3 Prevents and Reverses Myofibrillar Myopathy through Mechanosignaling Restoration.

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Title: AAV-Delivered RNAi Targeting Mutant LDB3 Prevents and Reverses Myofibrillar Myopathy through Mechanosignaling Restoration.
Authors: Pathak P; Neurogenetics Branch, National Institute of Neurological Disorders and Stroke (NINDS), NIH, Bethesda, Maryland, USA., Palmeri J; Neurogenetics Branch, National Institute of Neurological Disorders and Stroke (NINDS), NIH, Bethesda, Maryland, USA., Hale J; Neurogenetics Branch, National Institute of Neurological Disorders and Stroke (NINDS), NIH, Bethesda, Maryland, USA., Sabu-Kurian A; Neurogenetics Branch, National Institute of Neurological Disorders and Stroke (NINDS), NIH, Bethesda, Maryland, USA., Peiravi M; Murine Phenotyping Core, National Heart Lung and Blood Institute (NHLBI) NIH, Bethesda, Maryland, USA., Springer DA; Murine Phenotyping Core, National Heart Lung and Blood Institute (NHLBI) NIH, Bethesda, Maryland, USA., Li Y; Proteomics Core Facility, National Institute of Neurological Disorders and Stroke, NIH, Bethesda, Maryland, USA., Johnson KR; Bioinformatics Section, National Institute of Neurological Disorders and Stroke, NIH, Bethesda, Maryland, USA., Mankodi A; Neurogenetics Branch, National Institute of Neurological Disorders and Stroke (NINDS), NIH, Bethesda, Maryland, USA.
Source: BioRxiv : the preprint server for biology [bioRxiv] 2026 Mar 31. Date of Electronic Publication: 2026 Mar 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: AAV-Delivered RNAi Targeting Mutant LDB3 Prevents and Reverses Myofibrillar Myopathy through Mechanosignaling Restoration.
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  Data: <searchLink fieldCode="AU" term="%22Pathak+P%22">Pathak P</searchLink>; Neurogenetics Branch, National Institute of Neurological Disorders and Stroke (NINDS), NIH, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Palmeri+J%22">Palmeri J</searchLink>; Neurogenetics Branch, National Institute of Neurological Disorders and Stroke (NINDS), NIH, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Hale+J%22">Hale J</searchLink>; Neurogenetics Branch, National Institute of Neurological Disorders and Stroke (NINDS), NIH, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Sabu-Kurian+A%22">Sabu-Kurian A</searchLink>; Neurogenetics Branch, National Institute of Neurological Disorders and Stroke (NINDS), NIH, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Peiravi+M%22">Peiravi M</searchLink>; Murine Phenotyping Core, National Heart Lung and Blood Institute (NHLBI) NIH, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Springer+DA%22">Springer DA</searchLink>; Murine Phenotyping Core, National Heart Lung and Blood Institute (NHLBI) NIH, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Li+Y%22">Li Y</searchLink>; Proteomics Core Facility, National Institute of Neurological Disorders and Stroke, NIH, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Johnson+KR%22">Johnson KR</searchLink>; Bioinformatics Section, National Institute of Neurological Disorders and Stroke, NIH, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Mankodi+A%22">Mankodi A</searchLink>; Neurogenetics Branch, National Institute of Neurological Disorders and Stroke (NINDS), NIH, Bethesda, Maryland, USA.
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  Data: <i>Country of Publication: </i>United States <i>NLM ID: </i>101680187 <i>Publication Model: </i>Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>2692-8205 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2226928205%22">26928205 </searchLink><i>NLM ISO Abbreviation: </i>bioRxiv <i>Subsets: </i>PubMed not MEDLINE
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        Value: 10.64898/2026.03.28.715031
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        Text: English
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      – TitleFull: AAV-Delivered RNAi Targeting Mutant LDB3 Prevents and Reverses Myofibrillar Myopathy through Mechanosignaling Restoration.
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            – D: 31
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              Text: 2026 Mar 31
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