Potential to facilitate new discoveries in gene therapies.
By Raif Karerat
WASHINGTON, DC: A team, including two Indian American researchers, has developed a new user-friendly resource to accompany the powerful gene editing tool called CRISPR/Cas9, which has been widely adopted in the scientific community to make precise, targeted changes in DNA.
The breakthrough, which was facilitated by researchers from both Harvard University and the University of California, San Diego, has the potential to facilitate new discoveries in gene therapies and basic genetics research.
“We’ve taken a step towards making the CRISPR/Cas9 system more robust,” said Prashant Mali in a UCSD press release. Mali serves an assistant professor in the Department of Bioengineering at the UC San Diego Jacobs School of Engineering, and is co-first author of the study.
CRISPR/Cas9 is a relatively new genome engineering tool that can target a particular segment of DNA in living cells — such as a gene mutation — and replace it with a genetic sequence, according to NDTV.
The study describes an approach to simplify a laborious part of the gene editing process using the CRISPR/Cas9 system: choosing the best components to match specific gene targets.
“We built a computational model that accounts for all these different features. The end product is an interactive software for users to find guide RNAs that are predicted to be highly specific and highly active for their gene targets,” said Raj Chari, research fellow from the department of genetics at the Harvard Medical School.
The technology could help in finding gene therapies for genetic disorders such as cystic fibrosis and sickle cell anemia, said the team’s research, which was published in the journal Nature Methods.
“We hope to minimize the time and work in finding the most successful guide RNA sequence for a gene target, which will be helpful in finding new gene therapies,” stated Chari.