Gene Ther. 2026 Feb 28. doi: 10.1038/s41434-026-00603-7. Online ahead of print.
ABSTRACT
Thoracic aortic disease poses a significant threat due to its high mortality rates and genetic. underpinnings. While gene therapy holds promise for cures, the challenge lies in achieving. effective and safe gene delivery to the thoracic aorta. Tail vein injection (TI), is hindered by. off-target effects and hepatotoxicity, and traditional blinded percutaneous left heart injection. (TLI) carries a heightened risk of bleeding and mortality. To address these limitations. ultrasound-guided techniques present a viable solution. Adeno-associated virus (AAV) vectors. were delivered into the thoracic aorta of mice through TI, TLI, and ultrasound-guided. percutaneous left heart injection(ULI). While all three injection methods can achieve gene transduction in the thoracic aorta, the ULI approach provides the optimal balance between. high transduction efficiency and safety. The ULI method represents an efficient and safe. strategy for targeted gene delivery to the mouse thoracic aorta, providing a powerful tool for preclinical aortic gene therapy research that warrants broader application.
PMID:41764293 | DOI:10.1038/s41434-026-00603-7