A big worry of the CRISPR-Cas9 approach, in which the double-stranded DNA molecule is cut, is the cell’s response to that cut and how it is repaired.
With some frequency, this method leaves new mutations in its wake with unclear side effects.
The researchers prove that this system can be used in to treat various diseases in mice.
The main idea behind the Salk technique is the use of two adeno-associated viruses (AAVs) as the machinery to introduce their genetic manipulation machinery to cells in post-natal mice.
The complex lies in the DNA region of interest and fosters expression of a gene of interest.
Similar practices could be used to activate virtually any gene or genetic pathway while removing the risk of introducing potentially harmful mutations.
Initial data suggest that the technique is secure and does not produce unwanted genetic mutations. »