HOME >> BIOLOGY >> NEWS
Scientist who discovered Sly syndrome finds new research path to explore for treating the disease

ST. LOUIS -- Findings by a Saint Louis University research team led by the scientist who discovered Sly Syndrome 32 years ago point to a new direction for research into the rare genetic disorder that can cause bone deformities, vision and hearing loss, mental retardation and death in children.

The research is published in the Proceedings of the National Academy of Sciences Online Early Edition the week of Aug. 9.

"The importance of this research goes far beyond this rare disorder," said William Sly, M.D., chairman of the department of biochemistry and molecular biology at Saint Louis University School of Medicine. "It could potentially provide access to the brain for enzyme therapy in other similar diseases, most of which are more common than Sly Syndrome."

Sly Syndrome occurs in less than one in 250,000 births and is a progressive disorder that ranges in severity to extremely severe - resulting in death - to mild. It is caused by the deficiency of an enzyme called beta-glucuronidase, which leads to an accumulation of protein-sugar molecules known as mucopolysaccharides in many of the body's organs, including the brain. Enzyme replacement therapy - injecting the missing enzyme into the body - holds promise in treating physical problems caused by mucopolysaccharide accumulation in parts of the body other than the brain. The blood-brain barrier prevents the enzyme from reaching the brain.

But Saint Louis University researchers examining an animal model of Sly Syndrome previously had found that enzyme replacement therapy was effective in treating the brain if given while the mice were very young.

"Until now, the reason why the central nervous system of neonates, but not adults, responds to enzyme replacement therapy was unknown," said William A. Banks, M.D., an author of the article and a professor of geriatrics in the department of internal medicine and professor of pharmacological science at Saint Louis University S
'"/>

Contact: Nancy Solomon
solomonn@slu.edu
314-977-8017
Saint Louis University
9-Aug-2004


Page: 1 2 3

Related biology news :

1. Scientists to prototype cyberinfrastructure for research and education access to ocean observatories
2. Scientists sequence genome of kind of organism central to biospheres carbon cycle
3. Scientists find nanowires capable of detecting individual viruses
4. Scientists discover potential new way to control drug-resistant bacteria
5. Scientists explore genome of methane-breathing microbe
6. Scientists decipher genetic code of biothreat pathogen
7. Stuck on you: Scientists lay bare secrets of bacterial attachment proteins
8. Scientists discover proteins involved in spread of HIV-1 infection
9. Scientists fear new Ebola outbreak may explain sudden gorilla disappearance
10. Scientists reinvent DNA as template to produce organic molecules
11. Scientists visualise cellular handmaiden that restores shape to proteins

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:8/17/2018)... CASTLE ROCK, Colo. (PRWEB) , ... August 16, 2018 , ... ... for facioscapulohumeral muscular dystrophy (FSHD), announced that its Colorado Chapter will be holding its ... event, scheduled for September 8th at the Philip S. Miller Park, advocates for public ...
(Date:8/15/2018)... ... ... The Florida Chiropractic Association’s National Meeting on August 16th-19th in Orlando, ... U.S. will gain firsthand insights into the new laser’s advancements and superior treatment abilities. ... trial that has resulted in the granting of an FDA 510(k) market clearance for ...
(Date:8/14/2018)... ... 13, 2018 , ... NRD, LLC – NRD® is showcasing ... Gun. , The ionizing gun features ergonomic design and rugged construction. The ... and electropolished OSHA-compliant tip. This can be cleanroom packaged (P-2021-5502) or normally packaged ...
Breaking Biology News(10 mins):
(Date:8/31/2018)... ... 2018 , ... A recent expedition led by Dr. Blair ... of Industrial Science, the University of Tokyo, demonstrated how the use of autonomous ... of hard to reach deep sea ecosystems, like intermittently active methane seeps. Thanks ...
(Date:8/29/2018)... ... August 28, 2018 , ... CEO and founder of VetStem ... performed on an allogeneic (donor derived) stem cell product for osteoarthritis in canines ... in September. He is also the organizer of Breakout Session 4: Practitioner Primer ...
(Date:8/23/2018)... ... 22, 2018 , ... Crucial Data Solutions, Inc. announces the ... first fully-featured app for electronic data capture (EDC) and electronic participant reported outcomes ... percent of the market share for mobile devices worldwide; in turn, this enables ...
(Date:8/17/2018)... ... August 16, 2018 , ... ... pleased to announce the expansion of their team. Dr. Dawn Dufield, Ph.D., joins ... an innovative large molecule LC-MS/MS group that will specialize in developing quantitative LC-MS/MS ...
Breaking Biology Technology:
Cached News: