Gene therapy

Abstract

The human body is composed of a variety of proteins. Almost all human diseases are the results of improper production or function of proteins. Traditional small molecule drugs usually interact with proteins such as enzymes, hormones, and transcriptional factors to exert their therapeutic potential. However, many severe and deliberating diseases (e.g., 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.