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Stem Cell Therapy for Congenital Heart Disease: A Systematic Review
Diamantis I Tsilimigras1,2, Evangelos K Oikonomou3, Demetrios Moris2,4
1School of Medicine (D.I.T.).
Insights
Stem cell therapy shows promise for congenital heart disease (CHD) treatment, potentially improving cardiac function and quality of life alongside surgery. Further research is crucial to confirm efficacy and safety in well-designed clinical trials.
Area of Science:
- Regenerative Medicine
- Cardiology
- Biotechnology
Background:
- Congenital heart disease (CHD) is a common and diverse group of birth defects.
- While surgery is standard, stem cell therapy is emerging as a complementary or alternative treatment for specific CHD types.
- This study reviews current evidence and research on stem cell therapies for CHD.
Purpose of the Study:
- To systematically review published evidence and ongoing research on stem cell-based therapeutic strategies for congenital heart disease (CHD).
Main Methods:
- A systematic review of Medline, ClinicalTrials.gov, and the Cochrane library was performed.
- Searches included studies and their reference lists up to April 23, 2017.
Main Results:
- Nineteen studies were reviewed: 8 preclinical, 6 clinical, and 5 ongoing trials.
- Various stem cell delivery routes (intracoronary, intramyocardial, intravenous, epicardial) were reported.
- Preclinical studies often model right ventricle dysfunction and pulmonary hypertension; clinical trials primarily focus on hypoplastic left heart syndrome.
- Cell-based tissue engineering for self-growing grafts and patches is a recent development.
Conclusions:
- Stem cell delivery may offer benefits over surgery alone for CHD, improving cardiac function, growth, and quality of life.
- Encouraging preliminary results necessitate well-designed clinical trials to confirm efficacy and safety.
- Further laboratory research and translation to large clinical studies are essential for this potentially groundbreaking approach.
Background:
Congenital heart disease (CHD) constitutes the most prevalent and heterogeneous group of congenital anomalies. Although surgery remains the gold standard treatment modality, stem cell therapy has been gaining ground as a complimentary or alternative treatment option in certain types of CHD. The aim of this study was to present the existing published evidence and ongoing research efforts on the implementation of stem cell-based therapeutic strategies in CHD.
Methods:
A systematic review was conducted by searching Medline, ClinicalTrials.gov, and the Cochrane library, along with reference lists of the included studies through April 23, 2017.
Results:
Nineteen studies were included in this review (8 preclinical, 6 clinical, and 5 ongoing trials). Various routes of cardiac stem cell delivery have been reported, including intracoronary, intramyocardial, intravenous, and epicardial. Depending on their origin and level of differentiation at which they are harvested, stem cells may exhibit different properties. Preclinical studies have mostly focused on modeling right ventricle dysfunction or failure and pulmonary artery hypertension by using pressure or volume overload in vitro or in vivo. Only a limited number of clinical trials on patients with CHD exist, and these primarily focus on hypoplastic left heart syndrome. Cell-based tissue engineering has recently been introduced, and research currently is focusing on developing cell-seeded grafts and patches that could potentially grow in parallel with whole body growth once implanted in the heart.
Conclusions:
It seems that stem cell delivery to the diseased heart as an adjunct to surgical palliation may provide some benefits over surgery alone in terms of cardiac function, somatic growth, and quality of life. Despite encouraging preliminary results, stem cell therapies for patients with CHD should only be considered in the setting of well-designed clinical trials. More wet laboratory research experience is needed, and translation of promising findings to large clinical studies is warranted to clearly define the efficacy and safety profile of this alternative and potentially groundbreaking therapeutic approach.
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