ArticleMolecular therapy : the journal of the American Society of Gene Therapy2014
Targeting the splicing of mRNA in autoimmune diseases: BAFF inhibition in Sjögren's syndrome as a proof of concept.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Who cites it
13 citing papers in PubMed, 26 citations in OpenAlex.
- Exploring the role of alternative splicing in the pathogenesis of Sjögren's syndrome: identification of novel isoforms associated with salivary gland immune infiltration.Genes & genomics · 2025Article
- Risk Allele rs117026326-Mediated Alternative Splicing of GTF2I Promotes B Cell Proliferation in Primary Sjögren's Syndrome.Journal of immunology research · 2025Article
- Efficacy of BAFF inhibition and B-cell depletion in non-obese diabetic mice as a spontaneous model for Sjögren's disease.RMD open · 2024Article
- Get Spliced: Uniting Alternative Splicing and Arthritis.International journal of molecular sciences · 2024Review
- The decreased of peripheral blood natural killer cell is associated with serum IL-2 level in the renal tubular acidosis in patients with primary sjogren's syndrome.BMC immunology · 2023Article
- Identification of Key Genes for Pyroptosis-Induced Salivary Gland Inflammation in Sjogren's Syndrome Based on Microarray Data and Immunohistochemistry Analysis.Journal of inflammation research · 2023Article
- Alternative Splicing: A New Cause and Potential Therapeutic Target in Autoimmune Disease.Frontiers in immunology · 2021Review
- Transduction of Salivary Gland Acinar Cells with a Novel AAV Vector 44.9.Molecular therapy. Methods & clinical development · 2020Article
- Causal variants in autoimmune disease: a commentary on a recent published fine-mapping algorithm analysis in genome-wide association studies study.Annals of translational medicine · 2017Article
- Aquaporin gene therapy corrects Sjögren's syndrome phenotype in mice.Proceedings of the National Academy of Sciences of the United States of America · 2016Article
- Analysis of Associations of Human BAFF Gene Polymorphisms with Autoimmune Thyroid Diseases.PloS one · 2016Article
- Advances in salivary gland gene therapy - oral and systemic implications.Expert opinion on biological therapy · 2015Review
- Current and emerging treatment options for ANCA-associated vasculitis: potential role of belimumab and other BAFF/APRIL targeting agents.Drug design, development and therapy · 2015Review
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Authors and funding
11 authors at 8 institutions in 3 countries.
Funding
Abstract
BAFF (B-cell-activating factor of the tumor necrosis factor family), a pivotal cytokine for B-cell activation, is overexpressed by salivary gland (SG) epithelial cells in primary Sjogren's syndrome (pSS). ΔBAFF, a physiological inhibitor of BAFF, is a minor alternative splice variant of BAFF. A U7 RNA was reengineered to deliver antisense sequences targeting BAFF splice regions. A major decrease of BAFF messenger RNA (mRNA) and protein secretion, concomitantly with the increase of ΔBAFF mRNA, was observed in vitro. In vivo, SG retrograd instillation of nonobese diabetic mice by the modified U7 cloned into an adeno-associated virus vector significantly decreased BAFF protein expression and lymphocytic infiltrates and improved salivary flow. This study offers a rationale for localized therapeutic BAFF inhibition in pSS and represents a proof of concept of the interest of exon skipping in autoimmune diseases.
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