Featured Research

from universities, journals, and other organizations

Stem cells hold hope for Hurler’s syndrome

Date:
November 6, 2013
Source:
University of Adelaide
Summary:
Research using special adult stem cells is promising new hope for better treatments for the devastating genetic disease Hurler’s syndrome.

University of Adelaide research using special adult stem cells is promising new hope for better treatments for the devastating genetic disease Hurler's syndrome.

Hurler's syndrome has a frequency of one in 100,000 live births in Australia and sufferers develop severe mental and physical disabilities and often die in their early teens.

The disease is caused by a single defective enzyme that is essential for breaking down complex sugars in cells.

The researchers are modifying adult stem cells to make them produce large amounts of the deficient enzyme, and using them to replace cells which aren't functioning properly throughout the body. Preliminary results in laboratory studies are showing improvements in brain function.

"We have turned adult stem cells into little 'enzyme factories' by coupling them with a virus that makes them pump out high levels of the enzyme," says PhD candidate Matilda Jackson from the University's School of Molecular and Biomedical Sciences.

"Those stem cells can then be injected into the blood where they move around the body and become liver or bone or brain or other cells and start producing the missing enzyme. They automatically migrate to the areas of damage in the affected individual.

"So far in our laboratory studies we've measured improvements in brain function but we're yet to complete the analysis to determine if there are improvements in other organs."

Matilda is supervised by Dr Sharon Byers, Affiliate Senior Lecturer within the School of Molecular and Biomedical Sciences and Head of the Matrix Biology Unit at SA Pathology, based at the Women's and Children's Hospital.

"There are two current treatments for Hurler's syndrome ‒ costly enzyme replacement therapy or bone marrow transplants which require a perfectly matched donor," says Dr Byers. "And while they bring some improvement, neither of these treatments prevents damage to the brain and bones because not enough enzyme reaches either of these tissues.

"These stem cells, modified so they produce large quantities of the enzyme that people with Hurler's syndrome lack, offer great hope for a potential new therapy. If we can help reverse the disease symptoms, we could see these children able to perform normal tasks, giving them a better quality of life and increasing their life span."


Story Source:

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


Cite This Page:

University of Adelaide. "Stem cells hold hope for Hurler’s syndrome." ScienceDaily. ScienceDaily, 6 November 2013. <www.sciencedaily.com/releases/2013/11/131106084257.htm>.
University of Adelaide. (2013, November 6). Stem cells hold hope for Hurler’s syndrome. ScienceDaily. Retrieved August 20, 2014 from www.sciencedaily.com/releases/2013/11/131106084257.htm
University of Adelaide. "Stem cells hold hope for Hurler’s syndrome." ScienceDaily. www.sciencedaily.com/releases/2013/11/131106084257.htm (accessed August 20, 2014).

Share This




More Health & Medicine News

Wednesday, August 20, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Ebola-Hit Sierra Leone's Freetown a City on Edge

Ebola-Hit Sierra Leone's Freetown a City on Edge

AFP (Aug. 19, 2014) Residents of Sierra Leone's capital voice their fears as the Ebola virus sweeps through west Africa. Duration: 00:56 Video provided by AFP
Powered by NewsLook.com
101-Year-Old Working Man Has All The Advice You Need

101-Year-Old Working Man Has All The Advice You Need

Newsy (Aug. 19, 2014) Herman Goldman has worked at the same lighting store for almost 75 years. Find out his secrets to a happy, productive life. Video provided by Newsy
Powered by NewsLook.com
Researcher Testing on-Field Concussion Scanners

Researcher Testing on-Field Concussion Scanners

AP (Aug. 19, 2014) Four Texas high school football programs are trying out an experimental system designed to diagnose concussions on the field. The technology is in response to growing concern over head trauma in America's most watched sport. (Aug. 19) Video provided by AP
Powered by NewsLook.com
American Ebola Patient Apparently Improving, Outbreak Is Not

American Ebola Patient Apparently Improving, Outbreak Is Not

Newsy (Aug. 19, 2014) Nancy Writebol, an American missionary who contracted Ebola, is apparently getting better, according to her husband. The outbreak, however, is not. Video provided by Newsy
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:
from the past week

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