New gene editing technique changes DNA more safely and efficiently

Researchers at MIT (Massachusetts Institute of Technology) in the United States, developed a new technique CRISPR (English abbreviation for Set of Normally Spaced Short Palindromic Repetitions), editing large amounts of DNA at once, more secure and efficient. The work represents a significant advance in the field of biotechnology, with the potential to replace different problematic genetic sequences that could help treat a range of diseases.

Molecular editing method called PASTE (Programmable Insertion via Site-Specific Targeting Elements) uses the enzyme Cas9 to reach a specific defective gene and replace it with a new one, guided by a short RNA sequence to reach the correct part of the genome. The difference is that the process cuts one DNA strand and then the other instead of breaking both strands simultaneously, which in previous studies ultimately caused unwanted chromosomal rearrangements.

The promising technique aims to combat difficult-to-treat diseases such as cystic fibrosis and Huntington’s disease, as well as other blood diseases such as hemophilia and G6PD deficiency. Soon, researchers showed that new CRISPR based system it can even be applied to various human cells.

In lab tests, they inserted 13 different genes into 9 parts of the genome and achieved a 60% success rate. However, the PASTE efficacy rate was 2.5% in “humanized” mouse livers, consisting of approximately 70% human hepatocytes. The entire study was published in the scientific journal Nature Biotechnology and all genetic constructs are provided by the authors for use by other scientists.

“It’s great to think that other people can develop and apply these molecular technologies in ways we can’t even think of. It’s great to be a part of this emerging community,” said Jonathan Gootenberg, an MIT fellow involved in the study.