SCIENTIFIC EDUCATIONAL CENTER science idea

Bioengineers have repurposed the CRISPR system to make a smaller version of the genomic engineering tool. Its small size should facilitate delivery to cells, tissues and the human body for gene therapy.

The general definition of CRISPR gene editing systems is that they work like molecular scissors, cutting out selected sections of DNA.

However, many different CRISPR systems that are used or are undergoing clinical trials for gene therapy of eye, liver and brain diseases are still limited in their capabilities, because they all suffer from the same drawback: they are too large and, therefore, they are too difficult to deliver to cells, tissues or living organisms.

In an article published in Molecular Cell, scientists announce what they believe is an important step forward for CRISPR: an efficient, multi-purpose mini-CRISPR system.

While commonly used CRISPR systems-with names such as Cas9 and Cas12a denoting different versions of CRISPR – associated (Cas) proteins-consist of approximately 1000-1500 amino acids, the new "CasMINI" consists of 529.

During the experiments, the researchers confirmed that CasMINI can delete, activate and edit the genetic code, just like its more powerful counterparts. Its smaller size means that it will be easier to deliver to human cells and the human body, which makes it a potential tool for the treatment of various diseases, including eye diseases, organ degeneration and the treatment of genetic diseases in general.

The researchers have already begun to establish cooperation with other scientists to develop gene therapy.

They are also interested in how they can contribute to the development of RNA technologies – for example, what was used to develop mRNA vaccines against COVID-19 – where size can also be a limiting factor.

The study was published in Molecular Cell.

ab-news.ru