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SEND is a system for gene therapy from human proteins: a promising MIT project in precision and low reaction

The mRNA vaccines are demonstrating its effectiveness to combat the scourge of the coronavirus, presenting (without being perfect) a series of advantages that range from its effect to its manufacture. Hence, this technique to administer therapeutic or prophylactic agents represents a milestone in the history of science and an opening of possibilities to combat diseases such as malaria.

And along these lines, interesting paths are also opening up in order to achieve molecular therapies that are as precise as they are successful. Example of one of the last attempts in this field is el sistema SEND, with which an encapsulated RNA molecule can be “programmed” to be sent to cells and that could trigger a low or no immune response by its nature, they explain.

A programmable “messaging” for gene therapies that could lead to little reaction

The SEND system, which corresponds to Selective Endogenous eNcapsidation for cellular Delivery, is based on the use of natural proteins for the body in the capsule that houses the therapeutic mRNA molecule, such as the one that stimulates antibody production in the case of coronavirus vaccines. As detailed in the MIT publication, SEND includes the protein PEG10, a molecule that exists naturally in the body, but “tricking it”.

PEG10 binds to its own RNA to compose a protective capsule around it. What the researchers have done is redesign it so that selectively package other RNA and can deliver it, so they have been able to use SEND to in turn deliver the CRISPR (gene editing) system into mouse and human cells, so that they can edit specific genes.

The fact that SEND is made up of proteins that occur naturally in the body is key to less likely to trigger an immune response. But this has to be demonstrated with future studies, so that if it were so, it would open the door to create gene therapies that could be administered repeatedly without side effects.

SEND is the result of the work of researchers at MIT (the McGovern Institute for Brain Research), the Howard Hughes Medical Institute, the Broad Institute at MIT and Harvard University. For now has been tested in cellular models, in which it has proven to be successful, so it is not yet a proven tool in organisms and investigations are pending.

In that sense, given that PEG10 derived from a retrotransposon, genomic elements with self-amplifying properties (acting in a similar way to some viruses but completely implanted and adapted to our cells), the hope with SEND is also develop RNA transfer systems that already exist in the human body and that can be used for therapeutic purposes. There have already been precedents with another protein derived from a retrotransposon, ARC, but the packaging and transport that they have achieved with PEG10 was not achieved.

Gene therapies are complex and, although promising systems like this are developed, they require a lot of research and work to be applied. However, they show themselves as a hope against diseases such as Alzheimer’s or cancer among many others, so each step in the sense of making them possible is interesting and, in some cases, perhaps necessary.