The National Institutes of Health have awarded a $2.2 million grant to researchers at the Carl R. Woese Institute for Genomic Biology. The four-year grant will be used to develop more precise genome editing technologies for gene therapy applications.

Genomic editing has been a critical tool in furthering basic research and treating human diseases. Several of the tools, including the CRISPR-Cas9 system, have enabled researchers to modify the genomic DNA in living cells. However, the current approaches produce double strand breaks in the DNA, which can have undesired consequences. A newer class of DNA editing tools that can overcome these problems is prime editors, which can introduce changes in the DNA without causing double strand breaks.

Although prime editors are able to introduce several types of DNA changes, many challenges remain for their implementation in biomedical applications. The team has proposed to develop a toolset to overcome existing limitations and be able to edit target DNA sequences accurately in specific tissues in living mice, with a particular emphasis on the brain. They hope that this technology will be widely applied in the biotechnology and biomedical fields.

Gene therapies involving DNA editing technologies are poised to change the way that many diseases are treated, including some which were previously considered incurable. The new tools that will be created by this project could become the core components of new gene therapies for numerous disorders, such as Huntington’s disease or amyotrophic lateral sclerosis (ALS).  

The team includes Pablo Perez-Pinera (ACPP), an associate professor of bioengineering, Thomas Gaj (BSD), an assistant professor of bioengineering, and Jun Song (ACPP), a professor of physics.