Promises and Uncertainties of Gene Therapy and Genome Editing

Amy Tsurumi

Thursday, March 8 - March 29 10:00 a.m. - noon 4 sessions
The Engineering Center, One Walnut Street

This course will provide an overview of recent progress made in technologies  involving genetic manipulation for potential therapies – a concept that emerged  in the 1960s. Such novel methods provide promising avenues for treating severe genetic conditions for which there are currently no viable pharmacological options. However, despite having boundless prospects for breakthrough cures, the field is still at its infancy with looming ethical concerns that must be addressed.  

We will conduct case studies, including the controversial work by Dr. Stanfield Rogers, who, in 1970, attempted to cure hyperargininemia by viral injection; treated the first pediatric gene therapy patient at the NIH Clinical Center in 1990 for a severe immunocompromising disease; was responsible for the first documented death during a gene therapy clinical trial for a metabolic disease in 1999; and successfully treated the bubble boy disease in 2016.  We will also discuss current events, namely the FDA panel’s discussion of gene therapy for B-cell acute lymphoblastic leukemia and reporting of successful editing of a gene mutation related to hypertrophic cardiomyopathy in human embryos. We will also examine advances in genome editing methodologies, from early gene transfer attempts to the more recently adopted CRISPR/Cas9 system, with an emphasis on the science behind, the application of, the patent war surrounding, and the numerous start-up companies that  have emerged based on the latter. We will also survey the progression in policies and guidelines established by the NIH’s Recombinant DNA Advisory Committee and the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee.  

The format of the course will involve both lecture to provide scientific background knowledge relevant to the topics covered in lay terms, and active discussions based on assigned primary article readings from various major news agencies.

Teaching Style: Lecture with discussion    Weekly Preparation: 1-2 hours

    Amy Tsurumi

    Amy Tsurumi is a postdoctoral researcher at the Massachusetts General Hospital and a Beacon Hill resident. She received her PhD in genetics at the University of Rochester School  of Medicine & Dentistry and a Masters in Epidemiology at the Harvard T.H. Chan School  of Public Health. She has coauthored many scientific articles on epigenetic and genetic regulation of development, cancer, aging, infections, and burn trauma.