DNA editing technique provides new tool for disease research and gene therapy

Researchers at the University of Adelaide, the South Australian Health and Medical Research Institute (SAHMRI) and CSIRO have developed a new technique to edit genes in living cells with unprecedented efficiency.  

By modifying an existing CRISPR technology termed Prime Editing, Dr Fatwa Adikusuma and colleagues have developed a new tool to make DNA changes with up to 95% efficiency. The research is published in Nucleic Acids Research. 

“We are currently working on ways to use Nuclease Prime Editor for the treatment of genetic diseases such as blindness, muscular wasting and cystic fibrosis,”Professor Paul Thomas, Director, South Australian Genome Editing (SAGE) Facility, University of Adelaide

“This new technology, which we term Nuclease Prime Editor, provides a powerful tool to perform gene editing with high precision,” said lead author Dr Fatwa Adikusuma, a molecular biologist and genome editing expert at the University of Adelaide and South Australian Health and Medical Research Institute (SAHMRI) and a fellow of CSIRO Synthetic Biology Future Science Platform. 
 
“Nuclease Prime Editor could be an improved alternative for more efficient precise gene editing. Further optimisation of this tool could potentially bring this technology to the clinic for gene editing therapy to repair a wide array of disease-causing mutations.” 
 
Dr Adikusuma and Professor Paul Thomas, who was co-leader of the study, have been working on strategies to improve genome editing since CRISPR technology was first published almost ten years ago. Their team is currently working on new approaches to further enhance Nuclease Prime Editor for research and therapeutic applications. 
 
“Nuclease Prime Editor helps us to efficiently generate cell and animal models to investigate how genetic diseases are caused. We can also use these disease models to develop new therapies, including gene correction using the Nuclease Prime Editor itself,” said Prof Paul Thomas, Director of the South Australian Genome Editing facility which provides genetic mouse models for researchers.  
The study used Nuclease Prime Editor to rapidly generate a mouse model harbouring the most common mutation causing cystic fibrosis. 
 
“We are currently working on ways to use Nuclease Prime Editor for the treatment of genetic diseases such as blindness, muscular wasting and cystic fibrosis,” said Prof Thomas.