Date: 19.3.2018
UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called "nanospears" for the targeted delivery of biomolecules such as genes straight to patient cells. These magnetically guided nanostructures could enable gene therapies that are safer, faster and more cost-effective.
Gene therapy, the process of adding or replacing missing or defective genes in patient cells, has shown great promise as a treatment for a host of diseases, including hemophilia, muscular dystrophy, immune deficiencies and certain types of cancer.
Current gene therapy approaches rely on modified viruses, external electrical fields or harsh chemicals to penetrate cell membranes and deliver genes straight to patient cells. Each of these methods has its own shortcomings; they can be costly, inefficient or cause undesirable stress and toxicity to cells.
To overcome these barriers, Paul Weiss and Steven Jonas led a research team that designed nanospears composed of silicon, nickel and gold. These nanospears are biodegradable, can be mass-produced inexpensively and efficiently, and, because of their infinitesimal size - their tips are about 5,000 times smaller than the diameter of a strand of human hair - they can deliver genetic information with minimal impact on cell viability and metabolism.
Jonas compared the cutting-edge biomolecule delivery method to real-world delivery methods appearing on the horizon. Just as we hear about Amazon wanting to deliver packages straight to your house with drones, we're working on a nanoscale equivalent of that to deliver important health care packages straight to your cells.
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