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Study shows risks of using genome editing on embryos

2 November 2020
Appeared in BioNews 1070

A new study has concluded that using CRISPR-based genome editing on embryos makes unintended DNA changes that would be passed on to future generations.

US researchers made 40 embryos with eggs from healthy women and sperm from a man with a gene mutation that causes hereditary blindness. They then repaired the blindness-causing mutation using CRISPR/Cas9 genome editing, but instead of the mutation actually being corrected, the researchers found that about half of the embryos ended up missing large parts of the chromosome on which the gene was located, some even losing it entirely.

'If our results had been known two years ago, I doubt that anyone would have gone ahead with an attempt to use CRISPR to edit a gene in a human embryo in the clinic,' said lead author Dr Dieter Egli of Columbia University in New York.

The CRISPR genome editing approach has been praised for enabling researchers to make precise changes to the genome of cultured cells, living tissues, and animal embryos. Its use holds promise for treating disorders, and its discoverers won a Nobel Prize in chemistry last month (see BioNews 1067). The approach was also used in 2018 by now-imprisoned Chinese scientist Dr He Jiankui who controversially created the world's first genome-edited twin babies (see BioNews 1033).

Despite previous studies having shown CRISPR's ability to repair genetic defects in human embryos, Dr Egli warned that more extensive testing needs to be carried out to ensure other uncontrolled changes have not been made – like in this study.

'Our hope is that these cautionary findings should discourage premature clinical application of this important technology but can also guide responsible research to achieve its ultimate safe and effective use,' said Dr Egli.

The study was published in the journal Cell.

Allele-specific chromosome removal after Cas9 cleavage in human embryos
Cell |  29 October 2020
Lab tests show risks of using CRISPR genome editing on embryos
The Independent |  29 October 2020
Study identifies pitfall for correcting mutations in human embryos with CRISPR
Columbia University Irving Medical Centre |  28 October 2020
12 July 2021 - by Dr Dieter Egli 
I recently received a thoughtful letter from a college student I do not know – I'll call her Claudia* – who took the time to reach out to me with her perspective on heritable genome editing in embryos...
23 November 2020 - by Dr Patrick Foong 
CRISPR-based genome editing has revolutionised molecular life sciences, however the ISSCR provides guidelines for responsible and ethical research to achieve safe and effective use...
7 September 2020 - by Dr Jennifer Frosch 
Genome editing is not yet safe for creating germline changes in humans, an international commission has concluded...
29 June 2020 - by Dr Jennifer Frosch 
CRISPR genome editing may result in unwanted heritable genetic changes, which could lead to long-term risks in a clinical context...
9 December 2019 - by Jakki Magowan 
The MIT Technology Review has released excerpts of unpublished research from Dr He Jiankui's manuscript that ignored ethical and scientific norms when creating the world's first gene-edited twins...
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