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Huntington's disease in mice partly reversed using CRISPR

26 June 2017

By Meghna Kataria

Appeared in BioNews 906

Eliminating the faulty protein that causes Huntington's disease goes some way to reversing disease progression in mice, a study has found.

Scientists used the genome editing technique CRISPR/Cas9 to 'switch off' the disease-causing gene in mice brains, alleviating many associated symptoms, though not to the level of healthy mice.

'The findings open up an avenue for treating Huntington's as well as other inherited neurodegenerative diseases, although more testing of safety and long-term effects is needed,' said Professor Xiao-Jiang Li at Emory University School of Medicine, USA, and senior study author. The work was published in Journal of Clinical Investigation.

Huntington’s disease is a genetic condition caused by a mutant form of the huntingtin gene (mHTT). It produces a toxic protein that accumulates in brain cells and causes progressive cognitive and motor decline, typically in mid-life, eventually leading to death.

The team of researchers used adult mice that contained the human form of the mutant gene, and were already showing signs of the disease. Using CRISPR/Cas9, they introduced genetic changes in an afflicted region of the brain that prevented further production of faulty protein. After three weeks, the mouse brains showed a reduction in the build-up of the mutant protein, and a concomitant improvement in their physical functions - although not to the levels of the control mice.

These observations highlight the ability of brain cells to repair themselves, the researchers believe.

Extending the results to human patients, the authors wrote: 'Thus, reducing mHTT expression in the brains of elder HD patients might still be effective in alleviating neurological symptoms.'

The scientists also sequenced the entire genome of the mice brain cells, which showed that genetic changes had predominantly occurred in the targeted gene. These results follow concerns about the safety and specificity of CRISPR/Cas9 genome editing raised recently, particularly incidences of unwanted mutations and changes in unintended parts of the genome (see BioNews 903).

Genetic Engineering and Biotechnology News | 20 June 2017
Journal of Clinical Investigation | 19 June 2017
Health Canal | 21 June 2017
Medical News Today | 20 June 2017
MD Magazine | 21 June 2017


11 December 2017 - by Martha Henriques 
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14 August 2017 - by Emma Lamb and Annabel Slater 
Scientists have repurposed CRISPR to target the repetitive RNA sequences responsible for several genetic diseases...

12 June 2017 - by Lea Goetz 
Scientists have identified the first promising biomarker for Huntington's disease (HD) that could be harnessed in a simple blood test to predict disease onset and progression...
05 June 2017 - by Charlotte Spicer 
CRISPR may introduce hundreds of unwanted mutations into the genome, a small study finds...
22 May 2017 - by Jennifer Willows 
A woman whose father has Huntington’s disease has won the right to sue his doctors for negligence, for failing to tell her...
23 January 2017 - by Annabel Slater 
The fourth session of the Progress Educational Trust's annual conference 'Rethinking the Ethics of Embryo Research: Genome Editing, 14 Days and Beyond' delved into the new genome-editing technique CRISPR/Cas9 and what it means for embryo research...
21 November 2016 - by Paul Waldron 
Scientists in the US have used a genome-editing technique to partially restore vision in blind rats...

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