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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

Regulation of Angiogenesis and Blood Supply

Regulation of Angiogenesis and Blood Supply

Rapidly dividing tumors, embryos, and wounded tissues require more oxygen than usual, lowering the oxygen concentration in the blood. At low oxygen or hypoxic conditions, an oxygen-sensitive transcription factor called the hypoxia-inducible factor 1 or HIF1 is activated. HIF1 is a dimeric protein of alpha (ɑ) and beta (β) subunits. Under optimal oxygen conditions, HIF1β is present in the nucleus while HIF1ɑ remains in the cytosol. HIF1ɑ is hydroxylated by prolyl hydroxylase and factor...

Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists

Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists

Endothelins (ETs) are potent vasoactive peptides critical in the human body's various physiological and pathological processes. One of the most promising therapeutic strategies for treating pulmonary arterial hypertension (PAH) involves counteracting the effects of these endothelins using a class of drugs known as endothelin receptor antagonists.
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme (ECE). Of...

Mechanism of Angiogenesis

Mechanism of Angiogenesis

Blood vessel formation starts early during embryonic development, around day 7. In the extraembryonic yolk sac, mesodermal precursor cells called hemangioblast proliferate and differentiate into angioblast. Angioblasts express vascular endothelial growth factor receptor 2 or VEGFR2, which binds VEGF-A, a proangiogenic factor, guiding blood vessel formation. VEGF signaling promotes angioblasts to form a blood island in the developing embryo. Angioblasts further differentiate, giving rise to...

Pharmacogenomics: Identification of New Drug Targets

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...

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Related Experiment Video

Updated: May 28, 2026

In Vivo Gene Transfer to the Rabbit Common Carotid Artery Endothelium

In Vivo Gene Transfer to the Rabbit Common Carotid Artery Endothelium

Published on: May 6, 2018

Gene therapy in vascular disease.

Fazilat Sedighiani¹, Sigrid Nikol

¹Department of Clinical and Interventional Angiology, ASKLEPIOS Klinik St. Georg, Lohmühlenstraße 5, 20099 Hamburg, Germany.

The Surgeon : Journal of the Royal Colleges of Surgeons of Edinburgh and Ireland

|November 2, 2011

Summary

Cardiovascular gene therapy for peripheral artery disease is safe but lacks consistent efficacy in clinical trials. Further research is needed to improve therapeutic angiogenesis for treating ischemic tissues.

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Last Updated: May 28, 2026

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Area of Science:

Cardiovascular Research
Gene Therapy
Regenerative Medicine

Background:

Peripheral artery disease (PAD) presents a significant unmet medical need, especially in advanced stages.
Therapeutic angiogenesis aims to regenerate blood vessels using gene therapy or stem cells to treat ischemic tissues.
Understanding the complex regulation of angiogenesis and arteriogenesis is key to developing novel treatment strategies.

Purpose of the Study:

To summarize the development of therapeutic angiogenic strategies using gene therapy for PAD.
To review current clinical trials investigating gene therapy for vessel regeneration and restenosis prevention.

Main Methods:

Review of Phase I, II, and III clinical trials.
Analysis of published data and relevant websites.

Main Results:

Phase II trials for therapeutic angiogenesis show variability in outcomes due to differing methodologies.
A large Phase III trial failed to confirm the efficacy of fibroblast growth factor 1 gene transfer for critical limb ischemia.
Current gene therapy approaches for PAD have demonstrated safety but inconsistent efficacy.

Conclusions:

Cardiovascular gene therapy, specifically therapeutic angiogenesis, is safe.
Randomized controlled trials have not consistently demonstrated the efficacy of therapeutic angiogenesis.
Further advancements are required to establish gene therapy as a consistently effective treatment for PAD.