Subscribe to the BioNews newsletter for free

Login
Advanced Search

Search for
BioNews

Like the Progress Educational Trust on Facebook


 


 

Gene 'patch' fixes deafness mutation in mice

11 February 2013

By Reuben Harwood

Appeared in BioNews 692

A short strip of genetic material has been used to prevent hereditary deafness in mice. In a mouse model of the human condition Usher syndrome, which affects hearing, vision and balance, a 'genetic patch' was able to bind to and correct the mutation responsible for the condition. This ensured the proper assembly of a protein vital for the ability to hear and maintain balance.

Dr Michelle Hastings, who led the study at Rosalind Franklin University of Medicine and Science, Chicago, USA said: 'A single dose of the drug ['genetic patch'] to newborn mice corrects balance problems and allows these otherwise deaf mice to hear at levels similar to non-Usher mice for a large portion of their life'.

Approximately one in 6,500 children in the UK are born with Usher syndrome, resulting in varying degrees of deafness and blindness. Type 1 Usher syndrome is due to a mutation in the gene, USH1C, which codes for the protein harmonin. The mutation causes the protein to be produced in a stunted form that is not fully functional. Harmonin is important for the development of sensory hair cells in the inner ear, which send electrical signals to the brain when they detect sound.

Researchers injected newborn Usher mice with a genetic sequence, called an antisense oligonucleotide (ASO), which specifically targeted the mutation in the USH1C gene. This partially restored normal harmonin production.

The treated Usher mice showed normal growth of sensory hair cells needed to detect low-range sound frequencies; this was less apparent for hair cells needed to detect higher frequencies. Measuring nerve responses to sound showed Usher mice who received the therapy were able to detect low frequency sounds to a similar extent as normal mice. These therapeutic effects lasted several months, but by six months the hearing ability of the treated mice began to diminish.

Dr Hastings' team found that timing was crucial – the mice needed to be treated within the first 10-13 days after birth in order for the treatment to show a significant effect. This suggests that in humans, who have a far longer gestation period, the therapy would need to be given whilst the baby is still in the womb and would therefore require a complex delivery system, reports the BBC.

Previously, stem cells have been used to try to cure deafness in mice (as reported in BioNews 558) by generating new inner ear hair cells. The current study, published in Nature Medicine, demonstrates that congenital deafness in mice can be temporarily overcome using gene therapy.

Dr Ralph Holme, head of biomedical research at Action on Hearing Loss, said: 'More research is now needed to understand how this new therapy could be used to treat this particular type of Usher Syndrome in humans and discover whether vision can also be rescued'.

SOURCES & REFERENCES
BBC News | 05 February 2013
 
Nature Medicine | 04 February 2013
 
Rosalind Franklin University of Medicine and Science (press release) | 04 February 2013
 
Medical News Today | 05 February 2013
 

RELATED ARTICLES FROM THE BIONEWS ARCHIVE

13 February 2017 - by Jamie Rickman 
An improved gene-therapy technique using a synthetic virus has restored the hearing of deaf mice up to the level of a whisper...
06 February 2017 - by Caroline Casey 
A novel gene therapy technique has partially restored hearing and balance in deaf mice...
13 July 2015 - by Dr Greg Ball 
A technique that delivers genes into the inner ears of mice has been used to restore hearing, marking the first time that gene therapy has been successfully used to treat deafness...
28 April 2014 - by Ruth Retassie 
Gene therapy, performed using cochlear implants, has regenerated auditory nerves in guinea pigs, a study reports...
17 June 2013 - by Rhys Baker 
A 'non-invasive' method for delivering gene therapy into the eye has been developed by US researchers...

08 October 2012 - by Dr Sarah Spain 
US researchers have identified a genetic mutation responsible for the hearing loss that occurs with the condition Usher syndrome type I...
17 May 2010 - by Kyrillos Georgiadis 
US Scientists have produced mouse inner ear hair cells using stem cells, paving the way for a cure for deafness and other related balance disorders...
12 April 2010 - by Dr Will Fletcher 
Scientists have discovered a gene mutation that can cause children to be born deaf...
20 April 2009 - by Dr Will Fletcher 
A link has been found between progressive hearing loss and a microRNA gene - the first time this type of gene has been implicated in any inherited disorder. The findings are the result of convergent research by scientists from the Wellcome Trust Sanger Institute in Hinxton, England, and...
06 April 2009 - by Ailsa Stevens 
Scientists based at Sheffield University, UK, have successfully created human inner ear cells from fetal stem cells, according to a study published in the journal Stem Cells last week. The ability to replace damaged or abnormal inner ear cells, using ones grown in the lab, could in...

HAVE YOUR SAY
Be the first to have your say.

You need to or  to add comments.

By posting a comment you agree to abide by the BioNews terms and conditions


- click here to enquire about using this story.

Published by the Progress Educational Trust

CROSSING FRONTIERS

Public Conference
London
8 December 2017

Speakers include

Professor Azim Surani

Professor Magdalena Zernicka-Goetz

Professor Robin Lovell-Badge

Sally Cheshire

Professor Guido Pennings

Katherine Littler

Professor Allan Pacey

Dr Sue Avery

Professor Richard Anderson

Dr Elizabeth Garner

Dr Andy Greenfield

Dr Anna Smajdor

Dr Henry Malter

Vivienne Parry

Dr Helen O'Neill

Dr César Palacios-González

Philippa Taylor

Fiona Fox

Sarah Norcross

Sandy Starr


BOOK HERE

Good Fundraising Code

Become a Friend of PET HERE and give the Progress Educational Trust a regular donation