Scientists one step closer to Huntington's treatment using CRISPR
The CRISPR gene editing system is still being perfected, but a new variant may have found a way to treat Huntington’s disease.
In its current state, Huntington’s disease is an incurable, hereditary neurodegenerative condition that gradually erodes a person’s ability to move, talk and think, but a new breakthrough offers a potential treatment.
In a paper published to Frontiers in Neuroscience, a team of researchers from the Institute of Bioorganic Chemistry in Poland revealed how, with a new variant of the gene editing CRISPR system, it could be possible to edit out the Huntington’s gene and effectively treat a patient.
While it has generated significant media coverage with the idea of being able to ‘snip’ out defective genes and replace them with healthy ones, CRISPR has shown itself to be less than accurate in some occasions, with unintended side effects to surrounding genes observed in a number of experiments.
However, the Polish team said that its new CRISPR approach is safer and more specific than previously used versions, based on the results when testing it out in cellular models from a Huntington’s patient.
This version of CRISPR was recently designed to act as a nickase – an enzyme that cuts just one DNA strand instead of two – which increases the precision with which CRISPR can edit specific sequences of DNA.
“We demonstrated that excision of the repeat tract with the use of a CRISPR nickase pair resulted in inactivation of the huntingtin gene and abrogation of toxic protein synthesis in cellular models of Huntington’s disease,” said Dr Marta Olejniczak, group leader of the study.
“Because CRISPR nickases are known to be safe and specific, our approach provides an attractive treatment tool for Huntington’s disease.”
A launch pad
For years, researchers have tried many methods to silence the defective gene that produces a toxic protein, progressively accumulating and damaging the patient’s neurons.
These attempts included trying to interrupt the production of the protein through DNA and RNA approaches, but none provided any beneficial results.
Since CRISPR’s introduction into genetics in 2012, however, there have been many looking to the technology to find a new way, and this latest breakthrough could be the launch pad for a future treatment.
Published at Mon, 26 Feb 2018 10:00:43 +0000