COLUMBUS, Ohio Scientists have designed a nanoparticle that appears to effectively deliver genetic material into cells with minimal toxic effects.
In lab experiments, the researchers have found that this device, a vector, is able to deliver DNA deeply enough into a cell to allow genetic material to be activated a critical step in gene therapy. This vector is between 2 and 10 times more effective than other experimental materials, according to the research.
Biomedical researchers continue to pursue gene therapy as a treatment option for a variety of diseases known to be caused by a genetic defect. That pursuit includes efforts to ensure the safety of the therapy and find the most effective way to deliver the genes.
In many experiments, deactivated viruses that retain their ability to infect other cells are used as vectors to deliver normal genes intended to replace, or turn off, defective genes. But because some of the viruses can generate an immune response that complicates the treatment, scientists also are pursuing nonviral vector techniques for gene therapy.
In this case, Ohio State University scientists combined two ingredients calcium phosphate and a lipid shell to create a nanoparticle that protects DNA during its journey to the cell and then dissolves to allow for gene activation in the target cell. Nano refers to the tiny size of the particle in question its general structure can be detected only by an atomic force microscope.
Calcium phosphate is a mineral found in bones and teeth. Lipids are fatty molecules that help maintain the structure of cell membranes. Alone, calcium phosphate is toxic and lipids get absorbed by cells. Together, they form a protective and inflexible structure that, thanks to complex chemical reactions, self-destructs once inside a cell.
"Our nanoparticle is a foreign body just like a viral vector is, but it has a self-destructive mechanism so it does not generate a st
|Contact: Chenguang Zhou|
Ohio State University