U.S. scientists have reported a way to make artificial virus-like vectors that can enter human cells to perform specific tasks, such as gene editing. This high-volume, customizable nanomaterial could hold promise for future subjects in gene therapy and customized medicine. The study was recently published in Nature Communications.
Viruses are highly efficient biological “machines” capable of rapidly replicating and assembling offspring. Natural human viruses such as lentiviruses have previously been engineered to deliver therapeutic DNA or RNA in animals, but their delivery capacity is limited and there are some safety concerns. Making viral mechanisms available to humans by creating artificial viral vectors programmed with therapeutic molecules can lead to beneficial repairs and help restore human health.
Venigalla Rao of the Catholic University in Washington, D.C., and colleagues devised a way to create artificial viral vectors (AVVs) from a class of viruses called T4 phages that infect bacteria. These AVVs have a large internal capacity and a large outer surface that programs and delivers therapeutic biomolecules. In a proof-of-concept experiment, the authors generated AVVs containing proteins and nucleic acids to demonstrate their use in genome modification. In the lab, the platform successfully delivers full-length dystrophin genes to human cells and performs various molecular manipulations to engineer the human genome. In addition, AVVs are inexpensive when produced in large quantities, and these nanomaterials can remain stable for months.
The authors conclude that although further work is needed to assess safety, this approach has shown future promise for the clinical treatment of human and rare diseases. (Source: Feng Weiwei, China Science News)
Structural features of a 120 x 86 nm phage T4 shell with near-atomic resolution (frontal view on the left; The image on the right shows a cross-sectional view of the empty interior). Image courtesy of Venigalla B. Rao
Related paper information:https://doi.org/10.1038/s41467-023-38364-1
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