Newswise — Shengdar Tsai, Ph.D., an assistant member of the Department of Hematology at St. Jude Children’s Research Hospital, has received a five-year $3 million grant from the National Institutes of Health’s Common Fund for his work on genome editing safety.

The grant was awarded through the Somatic Cell Genome Editing program, which supports research to remove barriers to adoption of genome editing for clinical care.

Tsai’s grant will fund research to better understand genome editing safety. A genome is an individual’s complete set of DNA, the molecules that carry the genetic information needed to assemble and sustain life. Inherited or acquired changes in DNA can lead to a variety of diseases, including cancer. One major concern about manipulating genetic material comes from unwanted or accidental genetic mutations.

To address those concerns, NIH has awarded 21 Somatic Cell Genome Editing grants over the next five years. The grants are awarded through the Somatic Cell Genome Editing program, which was launched in Jan. 2018.

“Human genome editing technologies have opened up many far-reaching possibilities to treat disease,” said Christopher Austin, M.D., director of the NIH’s National Center for Advancing Translational Sciences, which oversees the program. “The intent is that these newly funded initiative will help speed the translation of genome editing to the clinic and to a greater number of patients with what are currently intractable illnesses and conditions.”

Tsai’s laboratory at St. Jude focuses on genomics and genome editing technologies for therapeutics. His group will receive funding for his research project titled, “A novel human T-cell platform to define biological effects of genome editing.”

In addition to Tsai, the grant’s expert co-investigators include fellow St. Jude researchers Stephen Gottschalk, M.D., Giedre Krenciute, Ph.D. and Yong Chen, Ph.D., along with Samantha Maragh, Ph.D., of the National Institute of Standards and Technology and Jian Ma, Ph.D., of Carnegie Mellon University.

“Our goal is to develop a human T-cell based platform to interpret the biological consequences of genome editing, in order to illuminate approaches that work and are safe for patients,” Tsai said.

SCGE program participants will form a collaborative consortium to share discoveries and ideas to accelerate the translation of promising genome editing technologies.

“We are delighted to be a part of this new consortium,” Tsai said. “By improving state-of-the-art methods to interpret and predict the unintended effects of editing, we hope that together we will inspire confidence in the safety of the next generation of promising genome editing therapies.”