Many human diseases such as cystic fibrosis, muscular dystrophy, AIDS, and cancer are caused by disruptions to genes. Gene therapy is an emerging technology that aims to replace missing or defective genes within human cells to treat these conditions. However, it is currently still very risky and not widely tested on humans. Existing approaches to gene therapy use viruses, electric fields or harsh chemicals. These may pose a risk to the cell in terms of toxicity or undesirable off-target effects, as well as being expensive and potentially inefficient. Scientists are currently working on ways to make gene therapy safer, faster, cheaper and more efficient. One of the major challenges in delivering gene therapy is making sure the therapeutic agents can target the desired areas and enter the cells. The trick is to get the therapeutics to enter the cell without killing it or affecting its growth. A group of researchers at the University of California – Los Angeles have developed tiny ‘nanospears’ made of silicon, nickel and gold. Because the tips of these spears are so infinitesimally small they are able to penetrate the membrane of the cell without harming it. Nanospears aren’t new – other groups have worked on similar structures powered by nanoscale motors in order to propel the spears towards their target. However, these newly-developed spears are even better as they are magnetic, so scientists can use a magnetic field to guide the nanospears without having to use toxic chemicals. They are also bio-degradable and can be mass-produced, making them cheaply available on a large-scale.
To test how well these nanospears worked, the researchers used them to deliver a gene for green fluorescent protein – that’s right, it does exactly what it says on the label – into a culture of human cells. Approximately 80% of the cells produced a green glow, indicating the gene reached its target, and of those cells 90% survived. That’s a marked improvement over current forms of gene therapy. So far this technique has been used to deliver DNA to cell culture systems. Not only is this an extremely a useful tool for researchers, but it may also help speed up the delivery of gene therapy and immunotherapy. But that’s not all. These nanospears could be used to deliver more than just DNA, paving the way for new nanomedicine technologies that could be used for applications ranging from biological research to human medicine. The researchers are currently working on optimising the nanospears technology and hope that in the future it could be used to guide the delivery of gene therapies within a human patient.
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Emi Schutz Archives
March 2018
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