In a new breakthrough in gene therapies and basic genetics research, a team of Indian American researchers has developed a user-friendly resource to make the powerful gene-editing tool more friendly. The gene-editing technique known as CRISPR/Cas9 has been widely adopted to make precise, targeted changes in the DNA. This finding would facilitate new discoveries in the field.
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. It has two components, a short 'guide RNA' with a sequence matching a particular gene target, and a large protein called Cas9 that cuts DNA precisely at that target. The research team describes an approach to simplify a laborious part of the gene editing process using the CRISPR/Cas9 system by choosing the best components to match specific gene targets.
Prashant Mali, assistant professor (UC) Jacobs School of Engineering, said, "We have taken a step towards making the CRISPR/Cas9 system more robust."
Raj Chari, research fellow from the department of genetics at the Harvard Medical School, said, "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. 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."
The paper said, "This technology ultimately has applications in gene therapies for genetic disorders such as sickle cell anemia and cystic fibrosis." The research team believes that this will be a useful resource for the community towards designing improved genome engineering experiments.
The study is published in Nature Methods