Featured Research

from universities, journals, and other organizations

New therapy offers hope to spinal muscular atrophy patients

Date:
June 9, 2010
Source:
University of Sheffield
Summary:
Children who suffer from the devastating disease spinal muscular atrophy are set to benefit from a new breakthrough in therapy developments.

Children who suffer from the devastating disease Spinal Muscular Atrophy are set to benefit from a new breakthrough in therapy developments by researchers at the University of Sheffield.

The research, which was published June 9 2010 in Science Translational Medicine, has shown that a novel gene transfer system has the potential to provide an effective therapeutic treatment for SMA patients.

SMA is a devastating motor neuron disease which affects children. It is caused by an abnormal survival motor neuron (SMN) gene, which leads to a reduction of SMN protein levels. The disease is currently incurable, and patients often require prolonged medical care as no effective treatments to alleviate the condition, currently exist.

The research team at Sheffield, led by Professor Mimoun Azzouz, tested whether a novel gene replacement therapy through a single injection was the most efficient way to treat the disease in a group of mice. They found that the injection, which expressed the SMN gene, successfully restored the SMN protein to normal levels and alleviated symptoms in the SMA model.

The new technique has the potential to develop a simple injection, without any requirement for risky and costly surgical interventions, and has achieved the highest therapeutic effects reported in the field to date. This has significant implications for the future treatment of SMA.

Professor Mimoun Azzouz, from the University's Department of Neuroscience, said: "I am delighted by the outcome of several years of efforts to tackle this devastating disease. These results bring us one step closer to a successful gene therapy treatment for patients with SMA."


Story Source:

The above story is based on materials provided by University of Sheffield. Note: Materials may be edited for content and length.


Cite This Page:

University of Sheffield. "New therapy offers hope to spinal muscular atrophy patients." ScienceDaily. ScienceDaily, 9 June 2010. <www.sciencedaily.com/releases/2010/06/100609142253.htm>.
University of Sheffield. (2010, June 9). New therapy offers hope to spinal muscular atrophy patients. ScienceDaily. Retrieved October 20, 2014 from www.sciencedaily.com/releases/2010/06/100609142253.htm
University of Sheffield. "New therapy offers hope to spinal muscular atrophy patients." ScienceDaily. www.sciencedaily.com/releases/2010/06/100609142253.htm (accessed October 20, 2014).

Share This



More Health & Medicine News

Monday, October 20, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

How Nigeria Beat Its Ebola Outbreak

How Nigeria Beat Its Ebola Outbreak

Newsy (Oct. 20, 2014) The World Health Organization has declared Nigeria free of Ebola. Health experts credit a bit of luck and the government's initial response. Video provided by Newsy
Powered by NewsLook.com
Another Study Suggests Viagra Is Good For The Heart

Another Study Suggests Viagra Is Good For The Heart

Newsy (Oct. 20, 2014) An ingredient in erectile-dysfunction medications such as Viagra could improve heart function. Perhaps not surprising, given Viagra's history. Video provided by Newsy
Powered by NewsLook.com
Ebola Worries End for Dozens on U.S. Watch Lists

Ebola Worries End for Dozens on U.S. Watch Lists

Reuters - US Online Video (Oct. 20, 2014) Forty-three people who had contact with Thomas Eric Duncan, the first person diagnosed with Ebola in the U.S., were cleared overnight of twice-daily monitoring after 21 days of showing no symptoms. Rough Cut (no reporter narration). Video provided by Reuters
Powered by NewsLook.com
Fauci: Ebola Protocols to Focus on Training

Fauci: Ebola Protocols to Focus on Training

AP (Oct. 20, 2014) Dr. Anthony Fauci, head of the National Institute of Allergy and Infectious Diseases, says he expects revised CDC protocols on Ebola to focus on training, observation and ensuring health care workers are more protected. (Oct. 20) Video provided by AP
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins