Dr. Rafael Escandon provides a brief history of the scientific discoveries enabling gene replacement and gene editing to become useful and potentially transformative therapies. These profound innovations come with profound ethical considerations. Escandon has worked in global clinical research and development and patient advocacy in the biotechnology industry for nearly 30 years and is currently the Founder and Principal of DGBI Clinical Research and Ethics Consulting – a consultancy to patient advocacy groups and to the biotechnology industry.