Navigation Links
Potential drug target in sight for rare genetic disease
Date:11/14/2013

re won't be a cure yet, but hopefully we can develop drugs to treat the less severe forms of the disease."

Yin added: "If we can treat people in the early stages of the disease, we may be able to lessen the severity of the disease and the mental delays."

James said this paves the way for the possibility of an alternative to enzyme replacement therapy, which involves weekly injections of a needle between a protective membrane and the spinal cord. The treatment is both painful and expensive, costing about $450,000 a year per patient.

The U of A team has been collaborating with researchers from Simon Fraser University and the University of British Columbia on their work. Haiying Bie, a post-doctoral fellow from the U of A, was also a key member of the team.

"We're very excited about this discovery," said James. "We've all worked very hard."

Approximately 70 diseases relate to improperly functioning enzymes and collectively affect 1 in 5,000 people around the world. These diseases are caused by a deficiency in the lysosomal enzymes responsible for breaking down large molecules in the body such as those that make up bones, cartilage or fat.


'/>"/>

Contact: Raquel Maurier
raquel.maurier@ualberta.ca
780-492-5986
University of Alberta Faculty of Medicine & Dentistry
Source:Eurekalert

Page: 1 2

Related biology news :

1. Researcher finds potential new use for old drugs
2. Bold new partnership launches to harness potential of data scientists and big data
3. Research reveals new understanding, warning signs, and potential treatments for multiple sclerosis
4. Research points to potential window for treating CMV and preventing mother-to-child transmission
5. A potential new class of fast-acting antidepressant
6. Angel or demon: Can a potentially invasive plant bring a positive influence to a region?
7. UAlberta medical researchers discover potential new treatment for colitis
8. Vinyl flooring linked to potentially harmful substances at schools and daycare centers
9. The potential of straw for the energy mix has been underestimated
10. New potential for nutrient-rich prairie fruits
11. USC scientists ID protein that regulates cellular trafficking, potential for anti-cancer therapy
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/30/2014)... of America (ESA) has elected ten new Fellows of ... acknowledges outstanding contributions to entomology in one or more ... following Fellows will be recognized during Entomology 2014 -- ... November 16-19, 2014 in Portland, Oregon. , DR. ... Department of Plant, Soil and Entomological Sciences at the ...
(Date:7/30/2014)... Jackson Laboratory has received a pledge of $1 million ... name the Laboratory,s new Neurobehavioral Biometry Center. ... Center will enable the Laboratory to develop a greater ... the ultimate goal of accelerating the pace of research ... serve as a premier resource for the Laboratory and ...
(Date:7/30/2014)... changing climate threaten some of the world,s most ... establish seed collections to save the seeds in ... some genetic diversity., For decades, these seed collections ... a one-size-fits-all approach for how many seeds to ... regardless of species, pollination mode, growth habitat and ...
Breaking Biology News(10 mins):Entomological Society of America recognizes 2014 fellows 2Entomological Society of America recognizes 2014 fellows 3Entomological Society of America recognizes 2014 fellows 4Entomological Society of America recognizes 2014 fellows 5Entomological Society of America recognizes 2014 fellows 6Entomological Society of America recognizes 2014 fellows 7Entomological Society of America recognizes 2014 fellows 8Entomological Society of America recognizes 2014 fellows 9Entomological Society of America recognizes 2014 fellows 10Entomological Society of America recognizes 2014 fellows 11Entomological Society of America recognizes 2014 fellows 12Entomological Society of America recognizes 2014 fellows 13Entomological Society of America recognizes 2014 fellows 14Entomological Society of America recognizes 2014 fellows 15Entomological Society of America recognizes 2014 fellows 16Entomological Society of America recognizes 2014 fellows 17Rossi Foundation pledges $1M for JAX neurobehavioral research center 2Saving seeds the right way can save the world's plants 2
... New Geology articles posted online ahead of ... the Austrian Miocene; a "natural wind tunnel" experiment on ... in water; glacial moraines in Colorado; new topographic data ... from Earth,s mantle; and experimentally generated volcanic lightning. ...
... , Dec. 10, 2013 /PRNewswire-iReach/ -- Source Medical ... and billing services to outpatient surgery centers and specialty ... to the position of Vice President-Clinical Informatics within ... provide product development insight that draws upon her 30-plus ...
... members George Whitesides, Ph.D., and David Edwards, Ph.D., have ... (NAI) joining 141 other innovators elected this year. ... distinction accorded to academic inventors who have demonstrated a ... made a tangible impact on quality of life, economic ...
Cached Biology News:Volatile ecosystems, a natural wind tunnel, volcanic lightning, and stress & strain on Venus 2Volatile ecosystems, a natural wind tunnel, volcanic lightning, and stress & strain on Venus 3Volatile ecosystems, a natural wind tunnel, volcanic lightning, and stress & strain on Venus 4Volatile ecosystems, a natural wind tunnel, volcanic lightning, and stress & strain on Venus 5Volatile ecosystems, a natural wind tunnel, volcanic lightning, and stress & strain on Venus 6Volatile ecosystems, a natural wind tunnel, volcanic lightning, and stress & strain on Venus 7SourceMedical Announces Hiring of Clinical Expert for ASC Division 2SourceMedical Announces Hiring of Clinical Expert for ASC Division 3Wyss Institute at Harvard University announces election of 2 faculty to Natl Academy of Inventors 2Wyss Institute at Harvard University announces election of 2 faculty to Natl Academy of Inventors 3
(Date:7/30/2014)... Vala Sciences was recently awarded ... Agency (EPA). This new contract was awarded for ... tests designed to assess the effect of chemical ... connections between neurons (synapses), which are critical for ... by environmental pollutants likely contributes to a variety ...
(Date:7/30/2014)... (PRWEB) July 30, 2014 LayerBio, Inc., ... completion of its first round of seed financing from ... enable preliminary efforts to develop a novel sustained-release product ... of our investors and their commitment to this important ... LayerBio. "Ophthalmology is one of many areas in which ...
(Date:7/29/2014)... number of significant technological challenges in their quest ... created for the James Webb Space Telescope. , ... a scientist at NASA,s Goddard Space Flight Center ... microshutter arrays that is, those activated by ... as the current technology,s magnetically activated arrays. This ...
(Date:7/29/2014)... Human Longevity, Inc. (HLI), a genomics and ... the healthy, high performance human life span, today announced ... expert in machine learning and machine translation as the ... Google where he was Distinguished Research Scientist and the ... HLI Co-Founder and CEO, J. Craig Venter , ...
Breaking Biology Technology:U.S. Environmental Protection Agency (EPA) Awards Vala Sciences a Second ToxCast Contract 2U.S. Environmental Protection Agency (EPA) Awards Vala Sciences a Second ToxCast Contract 3Revolutionary microshutter technology hurdles significant challenges 2Revolutionary microshutter technology hurdles significant challenges 3Revolutionary microshutter technology hurdles significant challenges 4Franz Och, Ph.D., Expert in Machine Learning and Machine Translation, Joins Human Longevity, Inc. as Chief Data Scientist 2Franz Och, Ph.D., Expert in Machine Learning and Machine Translation, Joins Human Longevity, Inc. as Chief Data Scientist 3
... , , PALO ... biotechnology company developing antiviral therapies, announced today that it has ... (HCV) technology from Stanford University. This technology, discovered in the ... M.D., Ph.D., is focused on a variety of novel targets, ...
... , BRIDGEWATER, N.J., Aug. 4 With acne ... percent of people between the ages of 12 and 24, Dermik ... the BenzaClin(R) (clindamycin 1%-benzoyl peroxide 5% gel) carekit, a unique prescribing ... agent to their skin care regimen. , , ...
... , , SOUTHAMPTON, Pa., Aug. 4 ... or the "Company") BioMedical Systems Division has been contracted ... installation of one BARA-MED((R)) monoplace hyperbaric chamber. The chamber ... Ltd. , , Hospimedica International Pvt. ...
Cached Biology Technology:Eiger BioPharmaceuticals Acquires Exclusive License to Novel Hepatitis C Virus (HCV) Technology From Stanford University 2Sanofi-aventis U.S. Introduces BenzaClin(R) Carekit 2Sanofi-aventis U.S. Introduces BenzaClin(R) Carekit 3ETC Biomedical Announces Chamber Sale in India 2ETC Biomedical Announces Chamber Sale in India 3ETC Biomedical Announces Chamber Sale in India 4