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A gene editing tool used to treat progeria in mice

06-Jan-2021

Key points from article :

A new hope for treating children born with progeria.

In mice with progeria-causing mutation - a cousin of genome editor CRISPR corrected the DNA mutation.

Researchers used base editing, a DNA-changing method originally inspired by CRISPR.

First tested the approach in cultured cells from 2 progeria patients.

Packaged the base editor into adeno-associated viruses (AAVs) - delivery vehicle for gene therapies.

Injected these into young mice with the progeria mutation.

After 6 months between 20% & 60% of their bone, skeletal muscle, liver, heart, & aorta carried the DNA fix.

Aortas bore no signs of the fibrous tissue growth or loss of smooth muscle cells.

But some of the rodents eventually developed liver tumours due to AAV.

Research by Broad Institute of MIT and Harvard published in Nature.

Hope to launch a clinical trial in kids soon using this approach

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