Navigation Links
Chemical chaperone could open door to treatment of neurological disorder
Date:2/5/2008

>

Niemann-Pick type C disease is a recessive inherited trait that can originate in one of more than 200 different mutations in the NPC1 gene, which lies on chromosome 18. The mutations lead to production of abnormal NPC1 protein. Normally, NPC1 protein plays an essential role in moving cholesterol out of cells, and if it doesn't function, cholesterol and other lipids accumulate.

Most scientists assumed that Niemann-Pick type C mutations produced NPC1 protein that didn't work correctly. So when a routine test in the Ory lab of a mutated NPC1 protein showed that the protein was in fact active in living cells, the researchers did a double take.

"It is unequivocal that the mutation causes disease in human patients," says Ory, also associate professor of cell biology and physiology. "Yet the mutated protein seemed functional when we introduced it into cells."

When they looked for the explanation for this aberration, Ory and colleagues found that a small proportion of the mutant protein actually could do the job of normal NPC1 protein. It turned out that the mutation caused most newly minted NPC1 protein molecules to fold into the wrong shape or to assume their final shape slowly so that the cell's quality control checkpoints rejected them. But some of the mutant protein molecules assumed the correct shape and made it to their proper destination.

That suggested to the team that Niemann-Pick type C disease possibly could be treated with chemicals that assist the mutant proteins produced in patients. Ory refers to these as chemical chaperones and indicates this approach could help the large NPC1 proteins during the process of folding their long, complex chains so that more of the mutant proteins fold properly and pass through the cell's quality control checkpoints.

In collaboration with the National Institutes of Health Chemical Genomics Center, Ory will next screen more than 200,000 compounds to see which ones increase
'/>"/>

Contact: Gwen Ericson
ericsong@wustl.edu
314-286-0141
Washington University School of Medicine
Source:Eurekalert  

Page: 1 2 3

Related medicine news :

1. Chemical culprit in popcorn workers lung identified
2. American Chemical Societys Weekly Presspac -- Sept. 5, 2007
3. Rohm and Haas to Webcast Presentation at Credit Suisse Chemicals Conference
4. Chemical Diversity Initiates International Prostrate Cancer Discovery Partnership
5. Low Doses of Red Wine Chemical May Fight Diabetes
6. National Academies advisory: genes and toxic chemicals
7. Genomic technologies to identify toxic chemicals should be developed
8. Malaria No More and Sumitomo Chemical Help Protect 1.4 Million Kids from Malaria
9. American Chemical Societys Weekly Presspac -- Oct. 17, 2007
10. Researchers study potential health benefits of natural chemicals in muscadine grape seeds
11. Brains Reward Chemical May Help Spur Obesity
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Chemical chaperone could open door to treatment of neurological disorder
(Date:7/13/2014)... Epidemiologists forecast an increase in the diagnosed incident ... incident cases in 2013 to 11,568 diagnosed incident cases ... 4.35%, during the forecast period. In 2023, the US ... of aGVHD in the 6MM, with 4,989 diagnosed incident ... diagnosed incident cases of aGVHD, with 704 diagnosed incident ...
(Date:7/13/2014)... OH (PRWEB) July 13, 2014 Wright ... bellwether trial has been scheduled in July of 2015, ... Lipitor lawsuits are in the discovery process ... Richard M. Gergel is presiding over the multidistrict litigation ... filed on behalf of individuals who allege that they ...
(Date:7/13/2014)... 13, 2014 Hope Palliative & Hospice Care ... support group at the Tamarack Senior Living Community in Palatine, ... 10:30 A.M. on the 3rd Wednesdays of every month. ... the Tamarack Community located at 55 S. Greeley St. Palatine, ... and there is no cost for the group. , ...
(Date:7/13/2014)... Recently, iFitDress.com, one of the most popular suppliers ... added a new selection of beautiful evening dresses ... a promotion for these evening gowns. Now, everybody can ... providing thousands of graceful gowns in its online store. ... France, Italy and many other countries. They are highly ...
(Date:7/12/2014)... July 12, 2014 The Chicken ... the past five years, buoyed by stable demand for ... management system. “Eggs are a relatively affordable staple for ... egg consumption generally grows in line with the size ... McKitterick. Furthermore, demand for new specialty egg products with ...
Breaking Medicine News(10 mins):Health News:Graft-Versus-Host Disease Market (GVHD) Growing at 4.35% AGR to 2023 Says a New Report Available at ReportsnReports.com 2Health News:Graft-Versus-Host Disease Market (GVHD) Growing at 4.35% AGR to 2023 Says a New Report Available at ReportsnReports.com 3Health News:Graft-Versus-Host Disease Market (GVHD) Growing at 4.35% AGR to 2023 Says a New Report Available at ReportsnReports.com 4Health News:Lipitor Lawsuits Move Forward With Scheduling Of First Bellwether Trial 2Health News:Lipitor Lawsuits Move Forward With Scheduling Of First Bellwether Trial 3Health News:Lipitor Lawsuits Move Forward With Scheduling Of First Bellwether Trial 4Health News:Hope Hospice Announces Grief Support Group at the Tamarack Senior Living Community 2Health News:New and Beautiful Evening Dresses Unveiled by iFitDress.com 2Health News:Chicken Egg Production in Canada Industry Market Research Report Now Available from IBISWorld 2Health News:Chicken Egg Production in Canada Industry Market Research Report Now Available from IBISWorld 3
... ... announcing the launch of its new website, Herbal Fen Phen Lawyers ( http://www.herbalfenphenlawyers.com/ ). ... to Fen Phen, despite warnings from the FDA that herbal Fen Phen may be ... attacks, strokes or death. , ...
... efficient to use the drug cetuximab only in colorectal cancer ... a study published online August 7 in the Journal ... tumors harbored wild-type KRAS were found to have a higher ... by the National Cancer Institute of Canada Clinical Trials Group. ...
... BEIJING, Aug. 7 /PRNewswire-Asia-FirstCall/ -- ... CMED ), a leading China-based medical device,company that ... announced the appointment of PricewaterhouseCoopers ("PwC") as,the Company,s ... previous independent auditors, KPMG. , ...
... , Senate Finance Committee Urged Not to ... , WASHINGTON, Aug. 7 America,s local and regional ... a provision imposing a 20% co-pay on laboratory services under Medicare. ... system, this provision would shift billions of dollars in costs to ...
... , MORRISTOWN, N.J., Aug. 7 Watson Pharmaceuticals, ... announced today that one lot of 100 mcg/hr Fentanyl Transdermal System ... wholesalers and pharmacies. The recalled patches are from Lot Number ... Watson Laboratories, Inc. and distributed by Watson Pharma, Inc. The ...
... , NEW YORK, Aug. 7 ... the final results of the previously announced offers by its ... "Issuers"), to purchase for cash (i) any and all of ... Secured Floating Rate Notes due 2010 (the "Floating Rate Notes") ...
Cached Medicine News:Health News:Hissey Kientz Announces the Launch of Herbal Fen Phen Lawyers Website 2Health News:Hissey Kientz Announces the Launch of Herbal Fen Phen Lawyers Website 3Health News:Cost-effectiveness of cetuximab in metastatic colorectal cancer 2Health News:China Medical Technologies Announces Change of Independent Auditors 2Health News:Seniors: Get Your Checkbooks Ready 2Health News:Watson Announces Limited Recall Of Fentanyl Transdermal System 2Health News:Watson Announces Limited Recall Of Fentanyl Transdermal System 3Health News:Duane Reade Holdings, Inc. Announces Expiration and Results of Tender Offers for Senior Secured Floating Rate Notes and Senior Subordinated Notes and Solicitation of Consents to Related Indentures 2Health News:Duane Reade Holdings, Inc. Announces Expiration and Results of Tender Offers for Senior Secured Floating Rate Notes and Senior Subordinated Notes and Solicitation of Consents to Related Indentures 3
(Date:7/10/2014)... , July 10, 2014 CVS Caremark Corporation (NYSE: ... directors has approved a quarterly dividend of $0.275 (27.5 cents) ... payable on August 1, 2014, to holders of record on ... CVS Caremark is dedicated to helping people on their ... in the United States . Through the ...
(Date:1/15/2014)... 15, 2014 Most osteoporosis patients want a choice ... online survey* sponsored by Mission Pharmacal Company. The survey, ... Foundation online support community, revealed that 74 percent of ... in a form other than a pill or a ...
(Date:1/15/2014)... Texas , Jan. 15, 2014  Novation, the leading ... awarded several new IT value-added reseller (VAR) national contracts ... IT goods and services at lower costs. These include ... services such as initiative assistance and ongoing IT consulting ...
Breaking Medicine Technology:Mission Pharmacal Conducts Online Survey of Osteoporosis Patients 2Mission Pharmacal Conducts Online Survey of Osteoporosis Patients 3New Novation Information Technology VAR Contracts Offer Significant Savings to Hospitals 2
... , RYE, N.Y. , July 28 Curemark, ... on the treatment of neurological diseases, announced that Saint Peter,s University Hospital ... Phase III clinical trials for CM-AT , Curemark,s autism treatment.   ... Saint Peter,s is affiliated ...
... SEATTLE , July 28 HemaQuest Pharmaceuticals, ... for sickle cell disease, beta thalassemia and EBV-related cancers, ... to its Series B financing by new investor, ... B financing to $16M . The financing is ...
Cached Medicine Technology:Curemark Opens Autism Trials at New Jersey Site 2Curemark Opens Autism Trials at New Jersey Site 3HemaQuest Pharmaceuticals Raises Additional $4M to Bring Total to $16M in Series B Financing 2
... a national reference laboratory and a worldwide ... ARUP offers an extensive test menu of ... clinical and anatomic pathology. Owned by the ... more than half of the nation's university ...
... national reference laboratory and a worldwide leader ... offers an extensive test menu of highly ... and anatomic pathology. Owned by the University ... than half of the nation's university teaching ...
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
Medicine Products: