Genetic information on genes is often used to produce proteins needed to fight diseases.  When specific genes are missing or defective, the proteins will not be expressed correctly, causing certain inherited diseases such as cystic fibrosis and muscular dystrophy, as well as acquired diseases such as cancer.  Gene therapy is a technique that replaces these missing or defective genes with normal ones.  The aim of gene therapy is to use actual genes to help the patient's own cells express the needed protein.  This is a better, safer, and cheaper way of fighting the diseases than by treating the patient with recombinant proteins (proteins made by genetically altered microorganisms, such as bacteria, with the necessary gene on it).  Gene therapy may also help fight diseases that would not be affected by recombinant proteins.  Gene therapy is accomplished by way of vectors.  The vectors carry the gene and are either implanted into directly into the patient or by culturing cells from the patient in a lab, using a vector to put the gene in the cells, and then reimplanting the cells into the patient's body.  Most likely, a wide range of delivery technologies is necessary for the treatment of diseases to be effective.
 

back to pros
back to the table of contents

Created as part of a term project for SCEN103 at the University of Delaware  Comments, suggestions, or requests to aliz@udel.edu "http://www.udel.edu/physics/scen103/CGZ/therapy.html"  Last updated May 11, 2000.  Copyright Jen Franchino, Vinnie Verruto, Allison Zuckerbrow,  Jeff May, Univ. of Delaware, 2000