Enhanced gene editing tool developed by LVPEI and IGIB

The genome editing system has high specificity, extended flexibility for genome coverage and the LVPEI team has validated its applicability for precision mutation editing and correction, said the researchers in their study

Hyderabad: In a collaborative work, scientists from Hyderabad-based LV Prasad Eye Institute (LVPEI), CSIR- Institute of Genomics and Integrative Biology (CSIR-IGIB) and others have presented an enhanced CRISPR-Cas9 based genome editing system that is more precise and efficient than existing technologies.

The genome editing system has high specificity, extended flexibility for genome coverage and the LVPEI team has validated its applicability for precision mutation editing and correction, said the researchers in their study published in Nature Communications on June 28.

The IGIB team led by Dr. Debojyoti Chakraborty demonstrated the improved editing efficiency of a Cas9 protein from a bacterium called Francisella novicida (FnCas9). Through an elaborate genetic engineering process, the edit efficiency and fidelity of this protein (enFnCas9) was enhanced. This improved tool is better than other popular Cas9 proteins and has the potential to be developed into new diagnostics and therapeutics, a press release said.

LVPEI researchers led by Dr. Indumathi Mariappan, senior scientist, Centre for Ocular Regeneration, then explored the suitability of these enFnCas9 variants for therapeutic applications and isolated skin fibroblast cells of a patient with a rare form of retinal dystrophy, which leads to severe vision loss. “This is a proof-of-concept and shows that these novel and improved editing tools are one step closer on the long road to therapeutic applications,” said Dr. Mariappan.

“This study will advance the utilisation of such gene correction tools for a wide number of genetic disorders. At a time when the pace of clinical trials in this space is very high in the west, this Indian study will boost support from funders, regulators and stakeholders for patients waiting for cure for their ailments through CRISPR,” said Dr Chakraborty.