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        <title>PekingHSC: Missing Piece, Restored Hope: A New Era of Intelligent RNA Therapeutics (2026) - Project Promotion</title>
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        <description>Title: Missing Piece, Restored Hope: A New Era of Intelligent RNA Therapeutics Description: A missing room in a building can be rebuilt. But what happens when a region of the human chromosome disappears? Wolf-Hirschhorn syndrome (WHS), caused by a microdeletion on chromosome 4, results in insufficient expression of critical genes and severe developmental consequences. Inspired by the challenge of restoring what is lost, we developed a SINEUP-based therapeutic strategy to enhance protein production from remaining functional transcripts without altering the genome. By integrating artificial intelligence with programmable RNA design, we aim to create a scalable platform for treating microdeletion disorders and bring new hope to patients affected by haploinsufficiency. Wiki | Official Page</description>
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