Press release Netherlands Institute for Neuroscience

Curing genetic disease in human stem cells

21 February 2020

Whereas the CRISPR-Cas technology developed in 2012 cuts out a defect in a gene and replaces it with a new piece, the latest CRISPR technology works differently. The aim is to repair the error in the DNA without cutting it. This theoretically makes it a safer form of genetic editing. Scientists from Utrecht have shown for the first time that this technique can effectively and safely repair the DNA of stem cells derived from cystic fibrosis patients in the lab.

The results of this study were published in the scientific journal Cell Stem Cell on the 20th of February.

The new CRISPR enzyme variant developed in the United States in 2018, is a technique that works more precisely and makes fewer errors, explain researchers Maarten Geurts from the Hubrecht Institute and Eyleen de Poel from UMC Utrecht. Maarten: 'With the traditional form of CRISPR-Cas, a certain piece of DNA is cut away, after which the cell has to repair itself, hopefully by replacing it with a ‘good’ piece of DNA that is made in the lab and presented to the cell. With the new form of CRISPR-Cas, called base-editing, the mistake in the DNA is identified, but not cut and replaced; it is repaired on site.'

More information

Read the full article on the website of Hubecht Institute.

Hubrecht Institute

The Hubrecht Institute – an Academy research institute – focused on developmental and stem cell biology. It encompasses 20 research groups that perform fundamental and multidisciplinary research, both in healthy systems and disease models. Since 2008, the institute is affiliated with the University Medical Center Utrecht, advancing the translation of research to the clinic. The Hubrecht Institute has a partnership with the European Molecular Biology Laboratory (EMBL).

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