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.

Related Articles


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 January 25, 2015 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 January 25, 2015).

Share This


More From ScienceDaily



More Health & Medicine News

Sunday, January 25, 2015

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Disneyland Measles Outbreak Spreads To 5 States

Disneyland Measles Outbreak Spreads To 5 States

Newsy (Jan. 24, 2015) Much of the Disneyland measles outbreak is being blamed on the anti-vaccination movement. The CDC encourages just about everyone get immunized. Video provided by Newsy
Powered by NewsLook.com
Growing Measles Outbreak Worries Calif. Parents

Growing Measles Outbreak Worries Calif. Parents

AP (Jan. 23, 2015) Public health officials are rushing to contain a measles outbreak that has sickened 70 people across 6 states and Mexico. The AP&apos;s Raquel Maria Dillon has more. (Jan. 23) Video provided by AP
Powered by NewsLook.com
Smart Wristband to Shock Away Bad Habits

Smart Wristband to Shock Away Bad Habits

Reuters - Innovations Video Online (Jan. 23, 2015) A Boston start-up is developing a wristband they say will help users break bad habits by jolting them with an electric shock. Ben Gruber reports. Video provided by Reuters
Powered by NewsLook.com
This Is What It's Like To Date A Med Student

This Is What It's Like To Date A Med Student

BuzzFeed (Jan. 23, 2015) Dating is now speed-dating... or studying. Video provided by BuzzFeed
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