NIH Launches Gene Editing Research Program

Gene editing can benefit people with numerous diseases and conditions, but there are several barriers that prevent it from being widely adopted.

The National Institutes of Health (NIH) announced it is currently working to overcome these barriers by developing a research program to improve the delivery mechanisms for gene editing tools, develop new genome editors, further understand the safety and efficacy of gene therapy, and assemble a tool kit that represents the findings, according to a press release.

The program, Somatic Cell Genome Editing, will award researchers nearly $190 million from the NIH Common Fund over the next 6 years, according to the release.

A DNA mutation can spark the development of rare diseases or common disorders. This change can be inherited from parents or developed over an individual’s lifespan, according to the press release. Gene therapy edits these mutated genes to cure various conditions.

Somatic cells are the primary focus of the new research program, according to the NIH. Somatic cells are non-reproductive cells that do not pass genetic information on to the next generation. DNA changes made through somatic cell editing will, therefore, not be inherited by children.

Over the past decade, scientists have made it possible to change the DNA of living cells by changing the sequence of DNA in the genome, according to the NIH. The gene is split and modified with a different protein to create immunity towards a disease.

Although gene editing has captured the interest of many, it has faced significant challenges in clinics. The program’s goal is to overcome these challenges and increase clinician uptake of the approach, according to the NIH

“Genome editing technologies such as CRISPR/Cas9 are revolutionizing biomedical research,” said NIH Director Francis S. Collins, MD, PhD “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.”

CRISPR, the newest type of gene therapy, uses the protein Cas9 to remake a gene after it. CRISPR is more affordable than other types of gene therapy and it uses RNA, rather than DNA, making it more efficient and precise, according to the NIH.

With 3 new gene therapies gaining approval in 2017 and many more in the pipeline, the NIH noted that advancing gene editing technology is crucial.

Opportunities to fund the Somatic Cell Genome Editing research are expected to be announced within the next month, according to the press release.