Monday , March 8 2021

New technology based on molderers and magnets can help in the treatment of genetic diseases

A new technology that relies on viruses that infect moth and nanomagnets can be used to edit defective genes that lead to diseases such as the Serbian cell, muscle dystrophy, and cystic fibrosis.

Bioengineer The Gang Bao University Rice University combines magnetic nanoparticles with a virus container extracted from a particular moth to deliver CRISPR / Cas9 user loads that modify genes in a particular tissue or organ with spatial control.

Since magnetic fields are easy to manipulate and, unlike light, easily pass through the tissue, Bao and his colleagues want to use them to control the expression of viral useful loads in target tissues by activating a virus that is otherwise inactivated in the blood.

The research appears in Natural biomedical engineering. In nature, CRISPR / Cas9 stimulates immune microbial systems by recording DNA attackers. This gives the microbes the ability to recognize and attack attacking attackers, but scientists are trying to adapt CRISPR / Cas9 to repair mutations that cause genetic diseases and manipulate DNA in laboratory experiments.

CRISPR / Cas9 has the potential to stop a hereditary disease – if scientists can set up mechanisms for regulating the genome into the right cell within the body. But the obstacles on the road remain, especially in the delivery of useful genes for editing genes with high efficiency.

Baa said that it would be necessary to arrange the cells in the body for the treatment of many diseases. "But effectively delivering machines for regulating genes into target tissue in a body with spatial control remains a major challenge," Bao said. "Even if you inject a viral vector locally, other tissues and organs can get there, and that could be dangerous."

The delivery vehicle, developed by Bao's group, is based on the virus that infects Autographa california, known as alfalfa alfalfa, born in North America. The baculovirus cylinder bricklayer (BV), the part carrying the carrier of the virus, is considered to be large to 60 nanometers in diameter and 200-300 nanometers in length. That's enough to transport more than 38,000 base pairs of DNA, which is enough to supply more units to edit the gene in the target cell, Bao said.

He said that the inspiration for combining BV and magnetic nanoparticles comes from talking with Rice researcher and doctor Haibao Zhu, who learned about the virus during a post-doctoral stay in Singapore but did not know anything about magnetic nanoparticles until he joined the Bao Laboratory. Tim Rice had previous experience using iron oxide nanocells and an applied magnetic field to open the walls of the blood vessels enough to allow the passage of drugs of large molecules.

"We really did not know whether this would work for editing the genes or not, but we thought," it's worth a hit, "Bao said.

Researchers use magnetic nanoparticles to activate BV and deliver gene-editing useful loads only where needed. To do this, an immuno-systemic C3 protein that normally inactivates baculoviruses is used.

"If we combine BV with magnetic nanoparticles, we can overcome this using a magnetic field," said Bao. "The beauty is that when we deliver it, genetic regulation takes place only on the tissue or part of the tissue, where we apply the magnetic field."

The application of the magnetic field allows BV transduction, the process of delivery of useful material that introduces the gene for the treatment of the gene into the target cell. A useful load is also a DNA that encodes both the reporter gene and the CRISPR / Cas9 system.

In the tests, BV was loaded with green fluorescent proteins or luciferase of firearms. Cells with proteins are brilliantly bright under the microscope, and experiments show that the magnets were very effective at targeted delivery of BV burden to both cell cultures and laboratory animals.

Bao pointed out that his and other laboratories are working on the delivery of CRISPR / Cas9 with adeno-associated viruses (AAV), but said that the capacity of BV for the therapeutic load is about eight times higher. "However, it is necessary to make BV transduction into target cells more effective," he said.


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