Gene-Editing System Fights Liver Disorder

Researchers at MIT have announced that they have created a way to cure adult mice of a liver disorder by using a new gene-editing program called CRISPR. This program is able to cut out mutated DNA and replace it with a corrected sequence, effectively eliminating any harmful mutations in the patients genetic code. CRISPR is modeled after the cellular mechanisms that bacteria use to protect themselves against viral infection, and utilizes a DNA-slicing enzyme called Cas9 bound to an RNA guide strand. While implementing the Cas9 enzyme into the organism, scientists also deliver a DNA template strand, which aids the target cell in rebuilding the correct DNA sequence after the mutation has been cut out.

Any kind of technology that takes a step forward in the potential to cure harmful diseases is a step in the right direction. The concept of simply "cutting out" a harmful sequence and replacing it with the correct sequence seems like basic solution on paper, but the implementation of the technology needed to apply the correct slicing enzyme and also introducing a template DNA strand is much easier said than done. Hopefully this kind of genome editing makes its way to the human testing phase, as this could be a huge step in curing a number of genetic mutation diseases, such as hemophilia or Huntington's disease.

Link to the article found here.
More information about CRISPR and its use by bacteria can be found here.