“Genetic scissors” continue to make m talk. This time, researchers from University of California at Berkeley, with Japanese researchers, managed to cure mice with Duchenne myopathy using CRISPR/Cas9. And rar than using a modified virus to carry “scalpel” into cells, researchers used gold nanoparticles (works published in Nature Biomedical Engineering).
What is CRISPR/Cas9? It’s a DNA correction system. It includes a guide molecule to go to right place in genome ( genetic GPS of chisel), a correct copy of DNA segment that one wants to put in place of one that is mutated and that does not work, finally enzyme that will cut DNA strand. It is refore necessary to bring se three elements to heart of cell in its nucleus.
Golden Scissors
So far only one type of shuttle was available, a modified virus (inactivated). Researchers at Berkeley (some of whom have created a company, GenEdit) have just demonstrated that three key elements of CRISPR/Cas9 could reach genes aboard gold nanoparticles. The nanoscale cargo is covered with a special membrane that allows it to sneak into cell and n nucleus up to chromosomes. Injected with model mice of Duchenne myopathy, se “golden scissors” allowed a restoration of 5.4% of overall functioning of gene responsible for disease. Which allowed m to double ir scores in strength and agility tests.
One of drawbacks of “genetic scissors” is that y can become “frantic” and cut without stopping. Moreover, when transported by a virus, y can lead to inflammatory and immune responses. The researchers who conducted experiments with “golden scissors” claim that ir means of transport avoids se disadvantages.
