Using CRISPR technology, scientists from the Chinese Academy of Sciences (CAS) are said to have devised a new “controllable, reversible, and safer” gene-editing strategy.
According to an article published on Wednesday in the South China Morning Post newspaper, the Cas13d-N2V8 system demonstrated a considerable reduction in the amount of off-target genes and no observable collateral damage in cell lines and somatic cells, indicating its future potential.
According to the researchers, the novel strategy using the Cas13 enzyme to target RNA is safer because RNAs are transient molecules that only exist in the cell for a brief amount of time and are not integrated into the genome.
“The Cas13 gene editing approach is safer, and the effects are more manageable and short-lived,” said Yang Hui, the study’s corresponding author and a researcher at China’s CAS Centre for Excellence in Brain Science and Intelligence Technology.
CRISPR is used in this approach.
The method employs Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, which is a natural defense mechanism that helps bacterial cells to recognize and eliminate viruses that assault them and has become one of the most widely utilized gene-editing techniques in recent years.
In the most extensively used technology, CRISPR-associated protein-9 (Cas9) is an enzyme that can cut the two strands of DNA in the genome to add or remove material.
“Patents protect CRISPR-Cas9 and Cas9-based gene editing techniques. Other companies do not have the opportunity to build them “Hui told Bioworld, a research-focused WeChat official account.
“Because CRISPR-Cas13 systems are more particular and precise, they have a broader applicability reach.”
The enzyme’s capacity to break non-target RNAs, known as collateral cleavage, restricts its therapeutic utility.
“Cas13 can destroy both target and non-target RNAs at random,” the researchers wrote on the CAS website, making it difficult to plan studies and evaluate results when using Cas13.
However, he noted, “the CRISPR-based gene editing technology does not permanently affect the genome, and the results of editing are manageable, reversible, and safer.”
System for detecting side effects
Yang and colleagues revealed how they developed a technique for detecting Cas13 collateral effects in mammalian cells, which they subsequently used to generate a huge number of variations.
“In conclusion, the Cas13 variations we created with minimal collateral effect are predicted to be more competitive for in vivo RNA editing and future therapeutic applications,” the study’s authors concluded.
Previously, Feng Zhang, a gene-editing researcher at MIT and Harvard and a CRISPR pioneer, developed the Rescue platform, which employed Cas13 to alter RNA.
“We were able to fill a major gap in the toolkit by inventing this novel enzyme and combining it with the programmability and precision of CRISPR,” Zhang stated in 2019.
The findings were first reported in the peer-reviewed journal Nature Biotechnology.