Progress and prospects: Gene Therapy clinical trials (Part 2)

Abstract

This is the second part of a review summarizing progress and prospects in gene therapy clinical research. Twenty key diseases/strategies are succinctly described and commented on by leaders in the field. This part includes clinical trials for skin diseases, neurological disorders, HIV/ AIDS, ornithine transcarbamylase deficiency, α1- antitrypsin deficiency, haemophilia and cancer.