Gene Therapy

Stem for Life - Board of Trustees

Gene therapy is a innovative type of cell therapy used to prevent, treat, and even cure genetic diseases by changing the expression of one’s genes. While gene therapy holds great promise, clinical trials are in their infancy and current therapies are primarily experimental.

To reverse disease caused by genetic damage, researchers isolate normal DNA and package it into a vehicle known as a vector, which acts as a molecular delivery engine. Vectors composed of viral DNA sequences have been used successfully in human gene therapy trials.

Researchers are working to develop and optimize vectors (viral and non-viral). Nonviral gene delivery involves complexing DNA with an agent that allows it to enter a cell nonspecifically. DNA delivered in this manner is usually expressed for only a limited time because it rarely integrates into the host cell genome.

Digital 3d model of DNA structure