PI3K/AKT/mTOR axis in vascular malformations: from molecular insights to targeted clinical trials
Recognition of PI3K/AKT/mTOR dysregulation provides a rationale for pathway-directed treatment. Development of selective agents and rational combination strategies-guided by molecular profiling and validated in preclinical and clinical studies-will be essential to enhance efficacy while minimizing resistance and toxicity.
Authors
- Chen Hua (ORCID: https://orcid.org/0009-0008-8502-1068)
- Xiao-Xi Lin
- Yuan-Yang Zheng
Institutions
- Shanghai Ninth People's Hospital (CN)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-12-22
- DOI
- https://doi.org/10.1186/s13023-025-04115-2
- Primary Topic
- Vascular Malformations and Hemangiomas
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Shanghai Jiao Tong University
- School of Medicine, Shanghai Jiao Tong University
- Fundamental Research Funds for the Central Universities