Long-term therapeutic efficacy and safety profiles of hpCas13d RNA editing in treating early-onset hypertrophic cardiomyopathy
These results underscore the promising translational potential of AAV-hpCas13d in treating cardiovascular diseases and advancing in vivo gene therapy.
Authors
- Yingmei Lou
- Lijun Fu (ORCID: https://orcid.org/0000-0003-2501-3740)
- Lianpin Wu (ORCID: https://orcid.org/0000-0001-9971-2654)
- Xiaoling Guo (ORCID: https://orcid.org/0000-0002-9014-5304)
- Jun‐Tao Lin (ORCID: https://orcid.org/0000-0002-0492-1021)
- Dan Zhu (ORCID: https://orcid.org/0000-0002-5857-1899)
- Zilong Geng (ORCID: https://orcid.org/0009-0009-3140-1214)
- Xing Liu (ORCID: https://orcid.org/0000-0002-3255-5399)
- Bing Zhang (ORCID: https://orcid.org/0000-0001-8676-2425)
- Min Zhang (ORCID: https://orcid.org/0000-0003-1589-7773)
- Yuze Wang (ORCID: https://orcid.org/0000-0002-0500-3644)
- Junxiao Wang (ORCID: https://orcid.org/0000-0001-7462-2716)
- Zhizhao Guo
- Ping Yang (ORCID: https://orcid.org/0009-0003-3987-7467)
Institutions
- Shanghai Jiao Tong University (CN)
- Wenzhou Medical University (CN)
- Shanghai Chest Hospital (CN)
- Shanghai Children's Medical Center (CN)
- Second Affiliated Hospital & Yuying Children's Hospital of Wenzhou Medical University (CN)
Publication Details
- Journal
- Life Sciences
- Published
- 2024-10-17
- DOI
- https://doi.org/10.1016/j.lfs.2024.123144
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 0.00