Gene Therapy

Abstract

The human body is composed of a variety of proteins. Almost all human diseases are the results of improper production or functioning of proteins. Traditional small molecule drugs usually interact with proteins such as enzymes, hormones, transcriptional factors and even RNA molecules to exert their therapeutic potential. However, many severe and debilitating hereditary diseases (e.g., type I diabetes, hemophilia, cystic fibrosis) and several chronic diseases (e.g., hypertension, ischemic heart disease, asthma, Parkinson’s disease, motor neuron disease, multiple sclerosis) remain inadequately treated by the conventional pharmaceutical approaches. This chapter describes progress made over the years in repairing, turning-off or replacing dysfunctional genes with exogenous DNA as a novel approach to treat, cure, or ultimately prevent disease by changing the expression of a person’s genes.