Researchers say gene-editing technology can be reduced in size and delivered by a virus.

Chinese scientists have created a smaller version of the CRISPR gene-editing protein that they say is more effective and capable of being delivered by new means. 

The research, led by Hongjian Wang and his team at Wuhan University, has been published in the journal PLOS Biology.

CRISPR gene editing, a revolutionary technique in medical research, involves altering DNA to treat diseases by removing harmful genetic material. 

However, delivering the genetic tools precisely to the right cells in the body has been a significant challenge. 

Non-pathogenic viruses offer a promising solution for delivering CRISPR, but the CRISPR proteins have traditionally been too large to fit into these viruses, while the smaller versions lacked sufficient efficiency.

The team addressed this issue by engineering a naturally small CRISPR protein found in certain bacteria. 

They modified this protein, named EbCas12a, by changing one of its amino acids to enhance its gene-editing capabilities. 

This new version, called enEbCas12a, showed effectiveness comparable to the best existing CRISPR proteins when tested on cells in the lab.

A major innovation came when the researchers successfully packaged enEbCas12a into an adeno-associated virus (AAV), a common tool in gene therapy due to its safety and efficiency in delivering genetic material to cells. 

They designed enEbCas12a to target a gene linked to cholesterol and administered it to mice with high cholesterol levels. 

After a month, the mice that received the treatment had significantly lower cholesterol levels compared to those that did not.

“The novel compact enEbCas12a, along with its crRNA, can be packaged into an all-in-one AAV system for convenient gene editing in vitro and in vivo with high fidelity, which can be very beneficial for future clinical applications and more tool developments including all-in-one AAV-based multi-gene editing, base editing, primer editing, etc.,” the researchers explain.

This achievement is not just a technical triumph but a potential game-changer in the field of gene therapy. 

The ability to package a highly efficient CRISPR protein into a virus means that it could be used to treat a wide range of genetic diseases in humans. 

The experts say more research is needed to determine its effectiveness and safety in human patients, but the latest study represents a significant step forward in making gene therapy more practical and accessible.