Disrupting the LINC complex by AAV mediated gene transduction prevents progression of Lamin induced cardiomyopathy
oleh: Ruth Jinfen Chai, Hendrikje Werner, Peter Yiqing Li, Yin Loon Lee, Khaing Thet Nyein, Irina Solovei, Tuan Danh Anh Luu, Bhavya Sharma, Raju Navasankari, Martina Maric, Lois Yu En Sim, Ying Jie Loh, Edita Aliwarga, Jason Wen Long Cheong, Alexandre Chojnowski, Matias Ilmari Autio, Yu Haiyang, Kenneth Kian Boon Tan, Choong Tat Keng, Shi Ling Ng, Wei Leong Chew, Michael Ferenczi, Brian Burke, Roger Sik Yin Foo, Colin L. Stewart
| Format: | Article |
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| Diterbitkan: | Nature Portfolio 2021-08-01 |
Deskripsi
Mutations in the LaminA gene are the second most common inherited cause of Dilated Cardiomyopathy, a major form of heart failure. Here the authors show that disruption of the nuclear protein SUN1 in cardiomyocytes, by AAV mediated transduction of a SUN1 inhibitor, significantly suppress cardiomyopathy progression, providing a potential therapeutic route to treat this disease.