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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...

Targeted Cancer Therapies

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...

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Related Experiment Video

Updated: May 7, 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

Targeting endothelial cells by gene therapy.

Thierry Vandendriessche¹, Marinee K Chuah

¹UNIVERSITY OF BRUSSELS;

|September 21, 2013

Summary

Researchers developed lentiviral vectors for targeted gene delivery to endothelial cells. This advancement offers new possibilities for treating genetic disorders, cardiovascular conditions, and cancers.

Area of Science:

Biomedical Engineering
Molecular Biology
Gene Therapy

Background:

Endothelial cells play crucial roles in vascular health and disease.
Targeted gene delivery to endothelial cells remains a challenge in therapeutic development.

Purpose of the Study:

To design and validate lentiviral vectors (LVs) for specific gene delivery into endothelial cells in vivo.
To explore the potential of this technology for treating endothelial-related diseases.

Main Methods:

Development of novel lentiviral vector constructs.
In vivo administration of LVs in preclinical models.
Assessment of gene expression and cellular targeting in endothelial cells.

Main Results:

More Related Videos

Characterization of Blood Outgrowth Endothelial Cells (BOEC) from Porcine Peripheral Blood

Characterization of Blood Outgrowth Endothelial Cells (BOEC) from Porcine Peripheral Blood

Published on: January 6, 2022

Vascular Gene Transfer from Metallic Stent Surfaces Using Adenoviral Vectors Tethered through Hydrolysable Cross-linkers

Vascular Gene Transfer from Metallic Stent Surfaces Using Adenoviral Vectors Tethered through Hydrolysable Cross-linkers

Published on: August 12, 2014

Related Experiment Videos

Last Updated: May 7, 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

Characterization of Blood Outgrowth Endothelial Cells (BOEC) from Porcine Peripheral Blood

Characterization of Blood Outgrowth Endothelial Cells (BOEC) from Porcine Peripheral Blood

Published on: January 6, 2022

Vascular Gene Transfer from Metallic Stent Surfaces Using Adenoviral Vectors Tethered through Hydrolysable Cross-linkers

Vascular Gene Transfer from Metallic Stent Surfaces Using Adenoviral Vectors Tethered through Hydrolysable Cross-linkers

Published on: August 12, 2014

Successfully engineered LVs demonstrated specific gene delivery to endothelial cells.
Efficient transduction of target cells was observed in vivo.
The developed vectors showed potential for therapeutic gene expression.

Conclusions:

Lentiviral vectors can be designed for precise gene delivery to endothelial cells in vivo.
This technology holds promise for advancing gene therapy strategies for hereditary disorders, cardiovascular diseases, and cancer.