Gene Therapy Breakthrough Promises Deafness Cure

Wednesday, 06 Feb 2013 12:19 PM

 

Share:
  Comment  |
   Contact Us  |
  Print  
|  A   A  
  Copy Shortlink
Scientists using gene therapy have partially restored hearing and balance in profoundly deaf mice, according to a study published in the journal Nature Medicine.

The research, still in its early stages and restricted to lab animals, may open up new avenues for tackling Usher syndrome, an inherited form of human deafness that usually goes hand in hand with blindness.

Researchers led by Michelle Hastings at the Rosalind Franklin University of Medicine and Science in Chicago, Illinois, aimed at a gene called USH1C which has been implicated in the "Type 1" form of Usher syndrome.

USH1C controls a protein called harmonin, which plays a vital role in hair cells — the cells in the cochlea of the inner ear that respond to sound waves and send an electrical signal to the brain.

The team devised a tiny strand of genetic material called an antisense oligonucleotide to "switch off" a faulty version of the gene that produces truncated forms of the protein.

The therapy was injected in newborn mice that had been genetically engineered to have the mutation.

A single injection partially restored their hearing at very low frequencies, and also reduced head tossing, a behaviour caused by impaired balance.

"These effects were sustained for several months, providing evidence that congenital deafness can be effectively overcome by treatment early in development to correct gene expression," the study says.

After the experiment, the mice were dissected, and their cochleas were found to have grown some hair cells.

The success of antisense oligonucleotides adds a further weapon in the quest to overcome deafness.

Last month, doctors at the Massachusetts Eye and Ear and Harvard Medical School reported on a gene drug that transformed cells in the cochlea into hair cells.

In 2012, investigators at the University of California, San Francisco targeted a fix for a faulty version of a gene called VGLUT3. The gene controls a protein that is vital for hair cells to send the signals they pick up.
 

© AFP/Relaxnews 2014

Share:
  Comment  |
   Contact Us  |
  Print  
  Copy Shortlink
Around the Web
Comments
Please review Community Guidelines before posting a comment.
>> Register to share your comments with the community.
>> Login if you are already a member.
blog comments powered by Disqus
 
Email:
Retype Email:
Country
Zip Code:
 
Find Your Condition
You May Also Like
Around the Web
Most Commented

The information presented on this website is not intended as specific medical advice and is not a substitute for professional medical treatment or diagnosis. Read Newsmax Terms and Conditions of Service.

Newsmax, Moneynews, and Independent. American. are registered trademarks of Newsmax Media, Inc. Newsmax TV, NewsmaxWorld, NewsmaxHealth, are trademarks of Newsmax Media, Inc.

NEWSMAXHEALTH.COM
© Newsmax Media, Inc.
All Rights Reserved