A new method of genome editing may be superior to the CRISPR/Cas9 technique. Researchers at Stanford University School of Medicine were able to cure mice with hemophilia by inserting a missing gene that expresses for a blood-clotting factor.
Dr. Mark Kay and his team developed this novel process that does not require the insertion of promoters and an endonuclease enzyme for its delivery. These new advances are safer and improve the genes longevity after insertion. The team was able to insert the new gene by, “hitching the expression of the new gene to that of a highly expressed gene in the liver called albumin”.
Another method for genome editing is the CRISPR/Cas9 technique which has dramatically decreased time spent in the lab to modify genomes of bacteria and other laboratory organisms. Some issues with the CRISPR/Cas9 are its potential use on humans because Cas9 can cut the DNA in unexpected places. This could kill the cell or alter it. Another problem that may occur is the promoter of the newly introduced gene could disturb the expression of nearby genes, which would lead to cancerous growth.
The technique was shown to be therapeutic to newborn and adult mice. In newborns the blood-clotting factor was expressed between 7 and 20 percent of normal levels, which has been shown to be a sufficient amount to clot blood in prior studies. The technique also worked well with adult mice even though the gene was inserted in 1 of every 100 liver cells.
The team now plans on experimenting on mice with livers composed of both human and mouse cells, “a model which they recently were able to show may be a good surrogate to further predict what will happen in humans.”
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