An international team of scientists has made a major step forward in our understanding of how enzymes ‘edit’ genes,
paving the way for correcting genetic diseases in patients. Researchers at the Universities of Bristol, Münster and the Lithuanian Institute of Biotechnology have observed the process by which a class of enzymes called CRISPR – pronounced ‘crisper’ – bind and alter the structure of DNA.
The results, published in the
Proceedings of the National Academy of Sciences (
PNAS) today [26 May], provide a vital piece of the puzzle if these genome editing tools are ultimately going to be used to correct genetic diseases in humans.
CRISPR enzymes were first discovered in bacteria in the 1980s as an immune defence used by bacteria against invading viruses. Scientists have more recently shown that one type of CRISPR enzyme – Cas9 – can be used to edit the human genome - the complete set of genetic information for humans....
Professor Mark Szczelkun, from Bristol University’s School of Biochemistry, said: "
An important challenge in exploiting these exciting genome editing tools is ensuring that only one specific location in a genome is targeted. Our single molecule assays have led to a greater understanding of the influence of DNA sequence on R-loop formation. In the future this will help in the rational re-engineering of CRISPR enzymes to increase their accuracy and minimise off-target effects. This will be vital if we are to ultimately apply these tools to correct genetic diseases in patients. ”
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