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Updated: May 4, 2026

In Vivo Gene Transfer to the Rabbit Common Carotid Artery Endothelium
Published on: May 6, 2018
Current gene therapy trials for vascular diseases
Paavo J Halonen1, Jussi Nurro, Antti Kuivanen
1University of Eastern Finland, A. I. Virtanen Institute, Department of Biotechnology and Molecular Medicine , P.O. Box 1627, FIN-70211, Kuopio , Finland +358 40 355 2076 ; seppo.ylaherttuala@uef.fi.
Introduction:
In the previous gene therapy trials for vascular diseases, safety of the therapies has been demonstrated with some evidence for clinical benefits. In the future, it will be important to also test the potential clinical benefits of the treatments in randomized and controlled trials with sufficient numbers of patients.
Areas Covered:
This review covers 15 currently ongoing cardiovascular gene therapy trials that aim to treat coronary artery disease, heart failure and peripheral artery disease. This review summarizes current trials and their main features in the cardiovascular field.
Expert Opinion:
In the gene therapy trials for vascular diseases, some limiting factors are still present. The trials have enrolled mainly elderly and severely affected patients who might not have the capacity to respond optimally to the therapies. Also, major cardiac adverse events, major amputations, mortality and other very demanding hard clinical end points have been used in relatively small patient populations. Therefore, there is an urgent need to enroll less severely affected patients and to use more informative surrogate end points in the forthcoming clinical trials.
Insights
Cardiovascular gene therapy shows promise for vascular diseases, but future trials need larger, diverse patient groups and refined endpoints for clearer clinical benefit assessment.
Area of Science:
- Cardiovascular Gene Therapy
- Vascular Disease Treatment
- Clinical Trial Analysis
Background:
- Previous gene therapy trials for vascular diseases demonstrated safety and some clinical benefits.
- Further research requires randomized, controlled trials with adequate patient numbers to confirm efficacy.
Purpose of the Study:
- To review 15 ongoing cardiovascular gene therapy trials for coronary artery disease, heart failure, and peripheral artery disease.
- To summarize the key features of current cardiovascular gene therapy trials.
Main Methods:
- Systematic review of 15 active cardiovascular gene therapy clinical trials.
- Analysis of trial objectives, patient populations, and endpoints.
Main Results:
- Ongoing trials focus on coronary artery disease, heart failure, and peripheral artery disease.
- Current trials often enroll elderly, severely ill patients and use demanding clinical endpoints in small populations.
Conclusions:
- Gene therapy for vascular diseases faces limitations including patient selection and endpoint choices.
- Future trials should include less severely affected patients and utilize more informative surrogate endpoints for robust results.
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