In a major step towards gene therapy, scientists claim to have designed a new device -- a nanoparticle -- which can effectively deliver genes into cells with minimal toxic effects.
In laboratory experiments, a team at Ohio State University has found that this device, a vector, is able to deliver DNA deeply enough into cell to allow genetic material to be activated -- a critical step in gene therapy.
This vector is between two-and-a-half and 10 times more effective than other experimental materials, according to the scientists.
In fact, in their research, the 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.
Together, they form a protective and inflexible structure that, thanks to complex chemical reactions, self-destructs once inside a cell.
"What we do is encapsulate a calcium phosphate core inside the liposome. And when this calcium phosphate gets inside a cell and that environment becomes acidic, it gets dissolved and then the gene can be very effectively released into the cytoplasm and transported to the nucleus. That is the theory," said lead scientist Chenguang Zhou.
He added: "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 strong response from the immune system.
"The material we use is also biocompatible. Calcium phosphate is in our bones and the lipids we use are synthetic, but can be biologically degraded. That's why there is low toxicity."
The findings have been published in the 'International Journal of Pharmaceutics'.
