Gene Therapy

How does gene therapy work? All life contains genetic information in the form of DNA. DNA can be reproduced and passed on to subsequent generations. A gene is a stretch of DNA that contains the blueprint for the sequence of amino acids making up a particular protein. Proteins are vital because they are used in catalyzing various biochemical reactions, they act as messengers, they regulate cell growth, development and reproduction, transport oxygen in the blood …

…so attractive to gene therapy, that is, their ability to integrate genes into a host's chromosomes also poses a serious drawback. Scientists have no control over how many copies of the genes become integrated or where on the chromosome they insert. This means that the vector may become inserted within another gene, disrupting or altering its expression. Or a gene may integrate within the regulatory region responsible for cellular proliferation and lead to cancer growth.