Reducing Granules Without Splicing Restoration Alleviates RBM20 Cardiomyopathy
The findings of this study demonstrated that RNP granules serve as a critical driver for RBM20 cardiomyopathy, and reduction of RNP granules through treatment with ASOs is a possible therapeutic option for RBM20 cardiomyopathy in patients carrying <i>RBM20</i> genetic variants in the nuclear localization signal region.
Authors
- Mei Methawasin (ORCID: https://orcid.org/0000-0002-2509-1936)
- Julia Muldoon
- Yanghai Zhang (ORCID: https://orcid.org/0009-0003-5307-6148)
- Henk L. M. Granzier (ORCID: https://orcid.org/0000-0002-9516-407X)
- Wei Guo (ORCID: https://orcid.org/0000-0002-9158-8107)
- John E. Smith (ORCID: https://orcid.org/0000-0002-0888-1274)
- Zaynab Hourani
- Zachery R. Gregorich (ORCID: https://orcid.org/0000-0002-4013-4869)
- Chunling Liu (ORCID: https://orcid.org/0000-0003-2974-628X)
- Yaqin He
Institutions
- University of Wisconsin–Madison (US)
- University of Arizona (US)
- Wisconsin Division of Public Health (US)
- University of Missouri (US)
Publication Details
- Journal
- Circulation Research
- Published
- 2025-04-17
- DOI
- https://doi.org/10.1161/circresaha.124.324781
- Citations
- 6
- Primary Topic
- RNA Research and Splicing
- Type
- article
- Field-Weighted Citation Impact
- 4.84