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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...

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: Apr 29, 2026

Investigation of Beige Fat Biology and Metabolism Using the CRISPR SunTag-p65-HSF1 Activation System

Investigation of Beige Fat Biology and Metabolism Using the CRISPR SunTag-p65-HSF1 Activation System

Published on: January 6, 2023

Targeting adipose tissue via systemic gene therapy.

S M O'Neill¹, C Hinkle¹, S-J Chen²

¹Cardiovascular Institute, Translational Research Center, University of Pennsylvania, Philadelphia, PA, USA.

|May 17, 2014

Summary

This summary is machine-generated.

Researchers developed a novel gene therapy using adeno-associated virus (AAV) vectors to specifically target adipose tissue. This method successfully corrected a genetic disorder in mice, showing potential for treating metabolic diseases.

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Isolation, Expansion, and Adipogenic Induction of CD34+CD31+ Endothelial Cells from Human Omental and Subcutaneous Adipose Tissue

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Published on: July 17, 2018

Area of Science:

Metabolic Homeostasis and Gene Therapy
Adipose Tissue Biology and In Vivo Gene Transfer

Background:

Adipose tissue is crucial for metabolic regulation, but in vivo modulation of its function remains a challenge.
Existing gene transfer techniques lack specificity for adipose tissue.

Purpose of the Study:

To develop a systemic in vivo gene transfer method specifically targeting adipose tissue.
To demonstrate the therapeutic potential of adipose-targeted gene therapy in a disease model.

Main Methods:

Construction of adeno-associated virus (AAV) vectors with adipose-specific regulatory elements.
Intravenous administration of AAV vectors in adult mice to identify optimal serotypes (AAV2/8).
Incorporation and delivery of the leptin gene via AAV2/8 vectors into ob/ob mice.

Main Results:

AAV2/8 demonstrated superior targeting of adipose tissue.
Engineered vectors successfully restricted reporter gene expression to adipose tissue.
Leptin gene therapy in ob/ob mice led to weight loss, reduced hyperinsulinemia, and improved glucose tolerance.

Conclusions:

Systemic delivery of adipose-targeted AAV vectors can effectively replace deficient genes in adipose tissue.
This approach shows significant therapeutic potential for metabolic disorders linked to adipose dysfunction.
The developed method offers a promising experimental tool for studying and treating diseases of adipose tissue.