Targeted modification of cis-elements in the CUL3 gene to restore exon 9 inclusion for treating Gordon syndrome
Our findings highlight the therapeutic potential of AA(-9, -10)TT and A18G in CUL3-related Gordon syndrome, suggesting the targeted modification of cis-elements could be an ideal and universal strategy to develop treatments for splicing-related diseases.
Authors
- Xiangzhong Zhao (ORCID: https://orcid.org/0000-0002-8064-066X)
- Qian Tang
- Shanshan Lu (ORCID: https://orcid.org/0000-0002-5438-8050)
- Xiaomeng Shi (ORCID: https://orcid.org/0000-0002-0053-0429)
Institutions
- Qingdao University (CN)
- Affiliated Hospital of Qingdao University (CN)
- Qingdao Municipal Hospital (CN)
Publication Details
- Journal
- Human Genomics
- Published
- 2025-10-21
- DOI
- https://doi.org/10.1186/s40246-025-00820-y
- Primary Topic
- RNA Research and Splicing
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- National Natural Science Foundation of China
- Natural Science Foundation of Shandong Province
- Qingdao University