Navigation Links
Scripps research scientists shed light on potential treatment for Gaucher's disease
Date:5/9/2010

LA JOLLA, CA May 7, 2010 In findings that advance scientists' understanding of a whole class of inherited disorders, a team from The Scripps Research Institute has shed light on a mechanism that enables a potential treatment for Gaucher's disease and other lysosomal storage diseases.

The findings were published in an advance, online edition of the journal Nature Chemical Biology on May 9, 2010.

"This study is likely to motivate clinical trials for the treatment of neuropathic lysosomal storage diseases, including Gaucher's disease, where the current standard of care, enzyme replacement therapy, is ineffective," said team leader Jeffery Kelly, who is chair of the Scripps Research Department of Molecular and Experimental Medicine, Lita Annenberg Hazen Professor of Chemistry, and a member of the Skaggs Institute of Chemical Biology. "The research is especially promising because we enhanced the cellular folding and function of mutated lysosomal enzymes, whose deficient function is linked to lysosomal storage diseases, using two distinct categories of FDA-approved drugs that have been shown to be safe and effective for the treatment of high blood pressure and muscle spasms."

"We wanted to uncover general principles that could be applied to a variety of loss-of-function protein misfolding diseases," added Derrick Sek Tong Ong, a graduate student in the Scripps Research Kellogg School of Science and Technology who was first author of the paper. "This study reveals how we can enhance the capacity of the cellular machinery to fold and traffic a mutant enzyme, so that the protein can function better."

In the new paper, the team revealed how the widely available prescription drugs diltiazem, verapamil, and in some cases dantrolene, acted on cells from patients with Gaucher's disease, increasing calcium levels in a subcellular compartment called the endoplasmic reticuluma convoluted membranous sac within the cell where the foldi
'/>"/>

Contact: Keith McKeown
kmckeown@scripps.edu
858-784-8134
Scripps Research Institute
Source:Eurekalert

Page: 1 2 3 4

Related biology news :

1. Scripps Research team provides groundbreaking new understanding of stem cells
2. New Scripps Research and GNF study helps explain how we can sense temperatures
3. Scripps Research scientists reveal how genetic mutations may cause type 1 diabetes
4. New nano-tool synthesized at Scripps Research Institute
5. Scripps Research scientists solve mystery of fragile stem cells
6. Scripps research team reveals how an old drug could have a new use for treating river blindness
7. Scripps Research scientists find two compounds that lay the foundation for a new class of AIDS drug
8. Scripps Research scientists create new way to screen libraries of 10 million or more compounds
9. Scripps Research team wins global race to achieve landmark synthesis of perplexing natural product
10. Scripps Florida scientists show lifeless prions capable of evolutionary change and adaptation
11. Scripps research team develops technique to determine ethnic origin of stem cell lines
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/3/2014)... , Dec. 2, 2014 As part of ... Inception Technologies is pleased to announce the release of ... customers to collect the workforce data that they need. ... have been left by existing readers. Many such devices ... connectivity and modern technology. Older models force users to ...
(Date:11/21/2014)... 20, 2014 Strict laws against distracted driving ... the North American and European automotive sector towards gesture ... recognition systems that are intuitive and able to retrieve ... industry. New analysis from Frost & ... in Europe and ...
(Date:11/18/2014)... 18, 2014  The Secure Identity & Biometrics ... jointly announce the formation of The Airport Entry ... Identity and Biometric Entry and Exit Solutions ... official support from BORDERPOL, the international non-profit organization ... provide expertise regarding border security, traveler and migration ...
Breaking Biology News(10 mins):Inception Technologies to Release New Biometric Reader 2Gesture Recognition Intensifies with the Rise of In-car Smartphone Integration, Says Frost & Sullivan 2Gesture Recognition Intensifies with the Rise of In-car Smartphone Integration, Says Frost & Sullivan 3Gesture Recognition Intensifies with the Rise of In-car Smartphone Integration, Says Frost & Sullivan 4Identity Management, Document Authentication and Biometric Companies Form "The Airport Entry and Exit Working Group", Releasing the Framework to Help Guide the Department of Homeland Security and Congress Towards Solutions 2Identity Management, Document Authentication and Biometric Companies Form "The Airport Entry and Exit Working Group", Releasing the Framework to Help Guide the Department of Homeland Security and Congress Towards Solutions 3
... , This release is available in ... be planted in "bee pastures," floral havens created as ... to produce successive generations of healthy young bees. ... perhaps only a half-acre each--easy to tend, according to ...
... sector and an Austrian research institute are chipping in ... public biological databases in the world. Although the majority ... Foundation, The Arabidopsis Information Resource (TAIR) database is now ... researchers worldwide use it monthly to study everything from ...
... , The growing amount of human noise pollution in the ... to their death, according to new research from a UK-led ... for weeks at sea, baby tropical fish rely on natural ... and thrive. However, the researchers found that short exposure ...
Cached Biology News:Bee pastures may help pollinators prosper 2Private support helps public plant research 2Marine Pied Piper leads Nemo astray 2
(Date:12/24/2014)... , Dec. 23, 2014 Sigma-Aldrich ... that the waiting period under the Hart-Scott-Rodino Antitrust ... acquisition expired on December 22, 2014, thereby completing ... requirement for the acquisition of the Company by ... antitrust clearance satisfies another condition to closing the ...
(Date:12/22/2014)... (PRWEB) December 22, 2014 The ... global leader of technical training across the life sciences ... Data Management (SCDM) to provide the organization's members ... programs —providing access to the more than 350 sessions ... members with 10% off when registering for a ...
(Date:12/22/2014)... , Dec. 22, 2014  Alternative Energy ... that it has signed a letter of intent ... has developed and patented a nanotechnology-based development platform ... products that enable rapid on-site collection and testing ... and health issues in an immediate, non-invasive and ...
(Date:12/22/2014)... The American Journal of ... research, reviews and editorials addressing developments and pathways ... published a provocative article exploring the role of ... potential treatment of prostate cancer. , The ... the possibility that there could be a connection ...
Breaking Biology Technology:Acquisition of Sigma-Aldrich by Merck KGaA, Darmstadt, Germany, Receives U.S. Antitrust Clearance 2Acquisition of Sigma-Aldrich by Merck KGaA, Darmstadt, Germany, Receives U.S. Antitrust Clearance 3CfPIE Announces Partnership with the Society for Clinical Data Management 2CfPIE Announces Partnership with the Society for Clinical Data Management 3ALNE Announces Intention To Acquire BioTechPharma 2Prostate Cancer Experts Dissect the Role of Follicle-stimulating Hormone in the Development, Progression and Potential Treatment of Prostate Cancer 2Prostate Cancer Experts Dissect the Role of Follicle-stimulating Hormone in the Development, Progression and Potential Treatment of Prostate Cancer 3Prostate Cancer Experts Dissect the Role of Follicle-stimulating Hormone in the Development, Progression and Potential Treatment of Prostate Cancer 4
... assets to stimulate growth , ,MADISON - More than ... Tuesday to hear Madison Mayor Dave Cieslewiczs plan for the ... a model for a forward economy, a 38-page ... as a vision for how Madisons public and private sectors ...
... RedPrairie Corporation Friday announced the availability ... services designed to ensure major retailer and Department of ... to meet each companys RFID implementation environment and schedule. ... foundation for the three others, is RFID Data Manager. ...
... bacteria that causes tuberculosis (TB) and leprosy are notoriously ... held in check for the past 50 years by ... therapy. , ,Now, however, a new chink has been ... diseases difficult to treat. The discovery, reported today (May ...
Cached Biology Technology:Madisons visionary plan for a healthy economy 2Madisons visionary plan for a healthy economy 3RedPrairie announces RFID 360 compliance software 2UW discovery could lead to new TB treatments 2UW discovery could lead to new TB treatments 3
Request Info...
Form: Ready to use Applications: Immunohistology-frozen, Immunohistology-paraffin, Western Blot, ELISA...
... x H): 17 3/4" x 8 ... slidesTime: Programmable 0 - 100 hoursControl: Programmable ... steps 40 user programmable protocols 3 operating ... Rapid temperature ramp-up from 37 - 95C ...
Goat polyclonal to p27 KIP 1 ( Abpromise for all tested applications). Antigen: Synthetic peptide: C-KRPATDDS STQNK, corresponding to Internal sequence amino acids 153-165 of Human p27 KIP 1 ...
Biology Products: