ArticleJournal of dental sciences2026
Antisense oligonucleotides-mediated rescue of the
Article in Journal of dental sciences, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background/purpose: Amelogenesis imperfecta (AI) is a group of rare, inherited disorders characterized by abnormal enamel formation. While identifying the genes and mutations that cause the disease is important, understanding its molecular pathogenesis is essential to developing precision and personalized medicine, which aim to stop disease onset, slow its progression, and provide a range of treatment options. Previously, we identified mutations that affect the conserved alternative splicing of the Materials and methods: HEK293 cells were grown on cell culture dishes for an Results: The altered splicing pattern was corrected by applying antisense oligonucleotides Conclusion: To the best of our knowledge, this is the first report to use ASO to silence mutant transcripts to correct splicing mutations confirmed to be disease-causing. This study represents a small step towards genetic interventions for AI and may serve as a basis for regulating the expression of mutant transcripts in developing teeth for future human studies.
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