An alternative splicing modulator decreases mutant HTT and improves the molecular fingerprint in Huntington’s disease patient neurons
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DOI: 10.1038/s41467-022-34419-x
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- Pawel Lisowski & Selene Lickfett & Agnieszka Rybak-Wolf & Carmen Menacho & Stephanie Le & Tancredi Massimo Pentimalli & Sofia Notopoulou & Werner Dykstra & Daniel Oehler & Sandra López-Calcerrada & Ba, 2024. "Mutant huntingtin impairs neurodevelopment in human brain organoids through CHCHD2-mediated neurometabolic failure," Nature Communications, Nature, vol. 15(1), pages 1-27, 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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