In addition,
more precise genome editing may allay public fears, especially if the resulting plant or animal is not considered transgenic because no foreign genetic material is introduced.
Using the switchSENSE ® technology, scientists in the IMED Biotech Unit at AstraZeneca have developed a novel «guide» molecule that is capable of directing the Cas9 nuclease to sites
of precise genome editing.
This approach,
combining precise genome editing in vitro with effective application of engineered cells in vivo, could provide «significant benefits for the treatment of many human diseases,» the authors note.
KIM Jin - soo, Director of the IBS Center for Genome Editing and corresponding author of the two studies commented, «Since the two studies have proved the superior specificity of Cpf1, this new nuclease will be more widely used
for precise genome editing that does not produce any unintended mutations.
«Employing the latest technology, including CRISPR / Cas9 — a powerful new tool that enables scientists to
make precise genome edits — I believe we have the opportunity to make great strides toward developing a therapy specifically for Huntington's disease.»
The most radical news at the conference was the first reported use of CRISPR / Cas9 to
generate precise genome edits (changing only a single base pair) in two genes, not only in cell lines in vitro, but also in specific tissues of embryos in ovo.
In early 2011, as he explored other ways to achieve efficient and
precise genome editing at the Broad, Zhang attended a talk by a fellow scientist, who described a natural genetic toolkit that some bacteria use to fend off invading viruses.
The IBS Center for Genome Editing published two studies at once in Nature Biotechnology, showing Cpf1's superiority as
a precise genome editing tool with no unintended mutations.
This is extremely important for the future of avian genetic rescue;
precise genome editing is the exact process needed to successfully achieve de-extinction and genetic diversity enrichment.
Potentially any inbred, mutant, or transgenic mouse strain can be genetically modified using this technique, too, opening up nearly any established mouse model to
precise genome editing.
Cas9 is the endonuclease enzyme part of CRISPR / Cas9 system that cuts the DNA, while RNA is the CRISPR guide, directing the enzyme to specific sites in the genome so that
precise genome edits are possible.