New Gene Silencers Offer Hope for Hereditary Blood Disorders

[Sly Saint]

According to a recent study, chemical tags previously thought of as genetic clutter are potent gene silencers; eliminating them could lead to safer treatments for hereditary blood disorders. In addition to providing a safer means of treating genetic diseases like sickle cell disease, a new version of CRISPR technology created at UNSW Sydney demonstrates that chemical tags on DNA, which are sometimes dismissed as little more than genetic cobwebs, actively mute genes.
Scientists have debated for decades whether methyl groups, small chemical clusters that build up on DNA, are the real source of gene suppression or just debris that builds up in the genome where genes are disabled.

However, in a recent study published in Nature Communications, researchers at the University of New South Wales, in collaboration with colleagues in the US at the St Jude Children’s Research Hospital, have demonstrated that deleting these tags can turn genes back on, proving that methylation is not only associated with silencing but also directly causes it.

We showed very clearly that if you brush the cobwebs off, the gene comes on, and when we added the methyl groups back to the genes, they turned off again. So, these compounds aren’t cobwebs – they are anchors.

Merlin Crossley, Study Lead Author and Professor, University of New South Wales​

The big picture​

So far, all work to achieve this has been carried out in a lab on human cells in a test-tube at UNSW and in Memphis.

Study co-author Professor Kate Quinlan says the discovery is not only promising for people with Sickle Cell disease, but other genetic diseases where turning certain genes on or off by altering the methyl groups avoids having to cut DNA strands.

“We are excited about the future of epigenetic editing as our study shows that it allows us to boost gene expression without modifying the DNA sequence. Therapies based on this technology are likely to have a reduced risk of unintended negative effects compared to first or second generation CRISPR,” she says

New CRISPR Technique Could Unlock Safer Treatments for Inherited Disorders

A study has revealed that chemical tags once regarded as genetic clutter are in fact powerful gene silencers – and removing them could unlock safer treatments for inherited blood disorders.

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