Somatic Cell Genome Editing's Health Relevance

Many diseases are caused by harmful changes, or mutations, in a person’s DNA. These genetic diseases include common diseases such as cancer and diabetes. Most rare diseases are also caused by DNA mutations such as, Duchenne Muscular Dystrophy, Huntington’s Disease, and Cystic Fibrosis. While each of the many thousands of rare diseases affect a small number of individuals, millions of Americans live with rare diseases, many of which have no current treatment options available.

Recent scientific advances in correcting these DNA mutations have made the possibility of treating, and even curing, genetic diseases much closer to reality. Research in this technology, referred to as genome editing, has increased at a tremendous pace, and the first clinical trials to use genome editing in humans are already underway. However, to fully realize the vision of treating many genetic diseases, several obstacles need to be overcome. One significant challenge to treating specific diseases is that it will often require making changes to the DNA in the cells affected by the mutation. And there are a wide variety of cells - in different parts of the body - that can be compromised in different genetic diseases. Getting genome editing tools into the various cells of the body will require the development of delivery methods specialized to the target cell type and location within the body.

Because of this challenge, the SCGE program is funding multiple projects that focus on a variety of strategies for delivering genome editing tools to different cells within the body that can be affected by genetic diseases, as illustrated below.

Infographic showing SCGE delivery awards and which region of the body they plan to target

Each box contains the name(s) and NIH grant number(s) of SCGE-funded researchers who are developing methods for targeting genome editing tools to the cells of the indicated organs. The icons are indicative of the general strategy for targeting being used by each project; including using viruses (Asokan, Sontheimer, Deverman, Gao), synthetic particles (Chen, Gao), or red blood cells as carriers (Ghiran); or direct delivery of the genome editing molecules (Saltzman, Sontheimer, Gong, McCray).

 

This page last reviewed on October 18, 2018