Physical Wellness

Researchers Show How DNA is 'Edited' To Correct Genetic Diseases

By Kamal Nayan | Update Date: May 27, 2014 10:11 AM EDT

Researchers have reportedly discovered the process by which a class of enzymes called CRISPR bind and alter the structure of DNA. The finding is a major step forward in our understanding of how enzymes 'edit' genes. 

Researchers said findings "provide a vital piece of 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 defense used by bacteria against invading viruses. 

Recently researchers discovered that one type of CRISPR enzyme - Cas9 - can be used to edit the human genome - the complete set of genetic information for humans.

These enzymes have been tailored to accurately target a single combination of letters within the three billion base pairs of the DNA molecule. This is the equivalent of correcting a single misspelt word in a 23-volume encyclopedia, the press release added. 

"An important challenge in exploiting these exciting genome editing tools is ensuring that only one specific location in a genome is targeted," said Professor Mark Szczelkun, from Bristol University's School of Biochemistry, in the press release.

"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. "

The results of the study have been published in the Proceedings of the National Academy of Sciences (PNAS). 

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