"Science": Innovative mRNA delivery technology is expected to bring changes to gene therapy

Today, the top academic journal "Science" published a new paper from the team of scientist Professor Zhang Feng

The inspiration for this breakthrough technology comes from the human body itself

Four years ago, researchers discovered a protein derived from such elements, called ARC

Unfortunately, attempts to manipulate these proteins have not been successful

First, the team conducted a systematic search in the human genome to find out which proteins might have similar functions

Scientists have discovered that a protein called PEG10 has the most potential for success

Using experiments conducted in cell lines, the researchers found that the amount of PEG10 released by the cells was significantly higher than that of other proteins

Based on these findings, the researchers further developed a system called SEND, the full name is "Selective Endogenous eNcapsidation for cellular Delivery" system (Selective Endogenous eNcapsidation for cellular Delivery), and PEG10 is exactly the system Core

▲SEND system can be released from cells for gene therapy (Image source: MIT; Credit: McGovern Institute)

Specifically, the researchers identified some "signal" sequences from the mRNA encoding PEG10, which are the key to the recognition of PEG10 mRNA by its protein

Then, the researchers made additional modifications to the PEG10 protein, adding a class of fusogen that promotes cell fusion, allowing it to specifically target different cells, tissues or organs

Using this system, scientists successfully delivered the CRISPR-Cas9 gene editing system to human and mouse cells to edit selected genes

This technology is still in early development, but it has shown great potential

Note: The original text has been deleted

Reference materials:

[1] Michael Segel, et al.

[2] Scientists harness human protein to deliver molecular medicines to cells, Retrieved August 19, 2021, from https://