New delivery mechanisms for genetic therapy will do us good

Source– The post is based on the article “New delivery mechanisms for genetic therapy will do us good” published in the mint on 24th February 2023.

Syllabus: GS3- Awareness in the field of biotechnology

Relevance: Regulatory system of drugs in India

News- A biotech company called Aera Therapeutics has unveiled a type of protein nanoparticle that can be used to deliver all sorts of genetic medicines around the body.

What are the challenges associated with present genetic therapies?

The presently available genetic therapy technologies can only fix the genome in reachable parts of the body. Its reach is very limited. The liver, eyes and blood are the main places where cures might be possible.

These technologies largely rely on viral vectors and lipid nanoparticles. But they can only efficiently deliver to certain Zip codes. Lipid nanoparticles’ routes are largely limited to the liver and eyes.

These have other limitations, for example how much cargo they can hold. Some genes for fixing the diseases are too big to fit inside a virus. It can be tough to squeeze the instructions for making Crispr tools into a usable lipid nanoparticle.

What are some facts associated with the new protein nanoparticle unveiled by Aera?

It is capitalizing on a recent discovery about a class of human proteins that are relics of viruses that infected humans ages ago.

One of these proteins assembled into a protein shell of a virus particle that stored the RNA needed for making more copies of itself.

Massachusetts Institute of Technology scientist Feng Zhang saw in the discovery an opportunity to exploit the system to deliver genetic material of his choice.

His lab experimented on the human genome for other proteins that assemble into protective shells and probed whether they were capable of transferring RNA.

In 2021, they showed that one of the proteins, called PEG10 could be repurposed to deliver gene-editing tools. That work became the foundation for Aera.

So far, around 50 of these self-assembling proteins have been found. The protective shells these proteins form come in a range of sizes. It means that some might be better suited for

slipping across the blood-brain barrier.

They are also, in theory, adaptable. Scientists have gotten very good at engineering proteins to do specific jobs. So, it is reasonable to think Aera researchers could engineer the capsids to travel to specific organs or tissues.

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