🧬 RNA Trans-splicing: A Game-Changer for CTNNB1 Disorder!
📌 Description
RNA trans-splicing is an innovative gene therapy approach that repairs faulty RNA by swapping out defective segments for healthy ones. For patients with CTNNB1 disorder—a rare genetic condition caused by mutations in the CTNNB1 gene—this method offers precision, safety, and hope. By restoring β-catenin function, RNA trans-splicing could revolutionize the treatment of neurodevelopmental disorders.
🧠Understanding CTNNB1 Disorder
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Cause: Mutations in the CTNNB1 gene affecting brain development and cell signaling.
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Impact: Developmental delays, intellectual disabilities, and motor challenges.
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Rarity: Classified as a rare genetic disorder.
🔬 How RNA Trans-splicing Works
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Identify the defective RNA transcript.
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Replace the faulty section with the correct RNA sequence.
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Restore normal protein function, enabling healthy cell processes.
💡 Benefits of This Approach
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🎯 Targeted precision – Fixes RNA without altering the DNA.
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🛡 Lower risk – Minimizes off-target effects.
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⏳ Long-lasting effects – Potential for sustained therapeutic benefit.
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