An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion
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DOI: 10.1038/s41467-022-28653-6
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- Florian Krach & Judith Stemick & Tom Boerstler & Alexander Weiss & Ioannis Lingos & Stephanie Reischl & Holger Meixner & Sonja Ploetz & Michaela Farrell & Ute Hehr & Zacharias Kohl & Beate Winner & Ju, 2022. "An alternative splicing modulator decreases mutant HTT and improves the molecular fingerprint in Huntington’s disease patient neurons," Nature Communications, Nature, vol. 13(1), pages 1-14, December.
- Yuma Ishigami & Mandy S. Wong & Carlos Martí-Gómez & Andalus Ayaz & Mahdi Kooshkbaghi & Sonya M. Hanson & David M. McCandlish & Adrian R. Krainer & Justin B. Kinney, 2024. "Specificity, synergy, and mechanisms of splice-modifying drugs," Nature Communications, Nature, vol. 15(1), pages 1-13, December.
- Zachariah L. McLean & Dadi Gao & Kevin Correia & Jennie C. L. Roy & Shota Shibata & Iris N. Farnum & Zoe Valdepenas-Mellor & Marina Kovalenko & Manasa Rapuru & Elisabetta Morini & Jayla Ruliera & Tamm, 2024. "Splice modulators target PMS1 to reduce somatic expansion of the Huntington’s disease-associated CAG repeat," Nature Communications, Nature, vol. 15(1), pages 1-17, December.
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