The National Institutes of Health (NIH) will launch an effort aimed at removing barriers that slow the adoption of genome editing for treating patients. This program, Somatic Cell Genome Editing, plans to award researchers approximately $190 million over six years beginning this year, pending availability of funds. These researchers will collaborate to improve the delivery mechanisms for targeting gene editing tools in patients, develop new and improved genome editors, develop assays for testing the safety and efficacy of the genome editing tools in animal and human cells, and assemble a genome editing toolkit containing the resulting knowledge, methods, and tools to be shared with the scientific community. The program is funded by NIH’s Common Fund.
“Genome editing technologies such as CRISPR/Cas9 are revolutionizing biomedical research,” said NIH Director Francis S. Collins, M.D., Ph.D. “The focus of the Somatic Cell Genome Editing program is to dramatically accelerate the translation of these technologies to the clinic for treatment of as many genetic diseases as possible.”
Many rare diseases, as well as some common disorders, are caused by changes in a person’s DNA, either through changes inherited from parents or those that occur during a person’s lifetime. Advances in genome editing made over the past decade now make it possible to precisely change the DNA code inside living cells. Despite widespread interest and investment in this field, many challenges remain preventing broad adoption of this technology in the clinic.
Somatic cells are any of the non-reproductive cells of the body, i.e. the cells that do not pass DNA down to the next generation. By focusing on somatic cells, any changes to the DNA introduced by the genome editing therapeutics will not be inherited.