Artificial viruses for their use in gene therapies

Some of them were designed by a team of scientists from the Catholic University of America (CUA) in Washington, USA artificial viral vectors (AVV) from viruses that infect bacteria improve gene therapy processes. These customizable nanomaterials could evade the possible memory of our defenses against them and have greater capacity.

viruses are efficient biological machines capable of rapidly replicating and assembling offspring. Some human examples, such as lentiviruses, were previously designed to deliver therapeutic DNA or RNA to animals, but their capacity was limited and they posed various safety concerns.

They have shown for the first time that bacteriophage T4 can coat itself in lipid, which facilitates the transfer of vital drugs into human cells.

Exploiting viral mechanisms of AVV constructs programmed with therapeutic molecules could perform beneficial repairs help restore human health. This method can be performed at low cost and high yield. In addition, the nanomaterials remained stable for several months, according to a study published in The nature of communication.

Scientists created AVV with a virus called bacteriophage T4. These vectors have a large internal volume and large external surface area for programming and delivery of biomolecules in therapy.

Laboratorio de Venigalla Rao in Rao’s laboratory. Investigators: Wenzheng Guo, Xiaorong Wu, Rao and Jingen Zhu. / Patrick G. Ryan

Founding Director of the CUA Bacteriophage Medical Research Center, Venigalla Raostudies the therapeutic potential of a type of virus that cannot infect humans and many of which are part of the microbiome of a healthy body.

Rao and his group showed for the first time that bacteriophage T4 they can be coated with lipidan innovation that facilitates the transfer of vital treatments into human cells.

“The technology platform for T4 artificial viral vector (T4-AVV) can be applied. a wide range of genetic diseasessuch as sickle cell disease, muscular dystrophy; also in diabetes or cancer”, confirms Rao SINC. “We believe we have shown that there is a way to develop a gene therapy treatment based on safe and effective bacteriophageswith almost unlimited healing potential,” he adds.

In proof-of-concept experiments, the authors generated AVVs using charge of proteins and nucleic acids demonstrate its use in genome engineering. The platform was able to successfully deliver the full-length dystrophin gene into human cells in the laboratory and perform various molecular operations to reshape the human genome.

The authors/authors believe that this method may hold promise in clinical treatment of rare diseases, but that they must continue to work on their security assessment. “T4-AVV technology must continue to evolve primary human cells isolated from blood before going to the clinic. The technology will also be tested in animal models such as mouse and rhesus macaque,” ​​explains Rao.

Restrictions and next steps

One of the most difficult limitations to avoid in experiments live It will be immune response which the bacteriophage can produce.

In this regard, the expert emphasizes that “immune reactions to the vector are expected to occur, but it will be necessary to evaluate in future clinical trials how this will affect the therapy and how it will be necessary to modify it”.

Current research in gene therapy It can be classified into three main approaches, which are based on the following vectors or vehicles for treatment: adeno-associated virus and lentivirus, lipid nanoparticles, and synthetic nanoparticles. All of these treatments remain experimental.

“Real therapy is years awaybut this work provides a model to develop life saving treatments and cures“, emphasizes the head of the research. “What we are investigating is a type of molecular surgery that can repair the defect safely, precisely and generate therapeutic results.”

The ultimate goal, Rao concludes, is that unlike current small-molecule drugs that sometimes have to be taken for life, a future bacteriophage-based drug “could provide a cure within hours or days.”