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Epigenome editing - technology safer and reversible than CRISPR


Key points from article :

CRISPR snips DNA to alter its sequence - alternatively researchers are excited about tech that leaves a DNA sequence unchanged.

Drugs are unfocused, acting on many genes at once, which means they come with toxic side effects.

Study in mice suggests epigenome editing is a potentially safer, more flexible way to turn genes on or off than editing DNA.

This might be less likely to cause harmful off-target effects and can be reversed.

“Dead” version of CRISPR’s Cas9 protein, capable of homing in on a specific place in the genome but unable to cut DNA.

Sangamo Therapeutics reported that in mice, adeno-associated virus (AAV) can tamp down brain levels of tau, involved in Alzheimer’s disease.

The AAVs being tested by many groups are expensive, and is gradually lost in cells when they divide.

Alternatively, lipid nanoparticles  are employed to deliver mRNA for COVID-19 vaccines, silencing PCSK9 to influences cholesterol levels.

Tune Therapeutics and Chroma Medicine are hoping to commercialise epigenome editors.

Leaves DNA sequence unaltered while retaining the ability to specifically home

Mentioned in this article:

Click on resource name for more details.

Chroma Medicine

Epigenetic editing company, therapy based on epigenetics

Fyodor Urnov

Scientific director for technology and translation, IGI, UC Berkeley

Jonathan Weissman

Professor at University of California San Francisco and Investigator at Howard Hughes Medical Institute

Sangamo Therapeutics

Company dedicated to advancing genetic therapies in order to treat patients living with serious genetic diseases.

Tune Therapeutics

Epi-therapeutic medicines working on epigenome editors