Chaperones News and Research

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People with Alzheimer's have lower ubiquilin-1 protein levels

Researchers have found that a protein variation linked by some genetic studies to Alzheimer's disease is consistently present in the brains of people with Alzheimer's.

2 Sep 2011

Scientists discover genetic switch to prevent neurodegenerative diseases

Scientists at Northwestern University report a surprising discovery that offers a possible new route for the treatment of neurodegenerative diseases. In a study of the transparent roundworm C. elegans, they found that a genetic switch in master neurons inhibits the proper functioning of protective cell stress responses, leading to the accumulation of misfolded and damaged proteins.

25 Aug 2011

CytRx sells global rights of molecular chaperone assets to Orphazyme

CytRx Corporation, a biopharmaceutical company specializing in oncology, today announced that it has sold the worldwide rights to its molecular chaperone assets to privately-held, Copenhagen, Denmark-based Orphazyme ApS.

17 May 2011

OncoGenex presents OGX-427 preclinical data in prostrate cancer at AACR meeting

OncoGenex Pharmaceuticals, Inc. announced today that preclinical data utilizing their pipeline compound OGX-427 was presented at the AACR 102nd Annual Meeting 2011.

5 Apr 2011

Protein folding may aid in developing new therapies for brain diseases

Misfold an origami swan and the worst that happens is you wind up with an ugly paper duckling. Misfold one of the vital proteins in your body - each of which must be folded in a particular way to perform its function - and the result can be a debilitating neurodegenerative disease such as Alzheimer's or Huntington's.

16 Mar 2011

FDA removes clinical hold for Amicus' AT2220 IND for treatment of Pompe disease

Amicus Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold for the AT2220 (1-deoxynojirimycin HCI) Investigational New Drug Application (IND). AT2220 is a pharmacological chaperone in development as a treatment for Pompe disease.

9 Mar 2011

Amicus to present additional positive data from Amigal Phase 2 extension study for Fabry disease

Amicus Therapeutics today announced that additional positive data from the ongoing Phase 2 extension study of its investigational drug Amigal™ (migalastat HCl) for Fabry disease will be presented at the Lysosomal Disease Network WORLD Symposium in Las Vegas, Nevada, February 16-18th, 2011.

17 Feb 2011

Acetylation complex fits like a halo over a histone

To understand the emerging science of epigenetics-a field that describes how genes may be regulated without altering the underlying DNA itself-scientists are deciphering the many ways in which enzymes act on the proteins surrounding DNA within cells.

21 Jan 2011

Scientists discover fundamental mechanism behind toxic protein aggregation in neurodegenerative diseases

Protein aggregation underlies several neurodegenerative diseases such as Alzheimer's, Huntington's chorea or Parkinson's. Scientists at the Max Planck Institute of Biochemistry (MPIB) in Martinsried near Munich, Germany, now discovered a fundamental mechanism which explains how toxic protein aggregation occurs and why it leads to a widespread impairment of essential cellular functions.

20 Jan 2011

Researchers identify gene mutation for that is essential for correct protein-processing in cells

A Jackson Laboratory research team led by Professor Patsy Nishina, Ph.D., has identified a mutation in a gene that's essential for correct protein-processing in cells. Defects in protein folding are associated with a variety of abnormalities and diseases.

10 Jan 2011

Researchers identify new mechanism that could help develop effective chemotherapy drug

Information on proteins is critical for understanding how cells function in health and disease. But while regular proteins are easy to extract and study, it is far more difficult to gather information about membrane proteins, which are responsible for exchanging elements essential to our health, like copper, between a cell and its surrounding tissues.

19 Oct 2010

Researchers develop KU-32 drug to prevent diabetic peripheral neuropathy

A drug developed at the University of Kansas has the potential to stop a debilitating condition of diabetes that often leads to pain in the extremities and even amputations, KU researchers have found.

13 Sep 2010

Clusterin associated with development, severity and progression of Alzheimer's disease: Report

Higher concentrations of clusterin, a protein in the blood plasma, appears to be associated with the development, severity and progression of Alzheimer's disease, according to a report in the June issue of Archives of General Psychiatry, one of the JAMA/Archives journals.

6 Jul 2010

ADDF awards $210,300 to Amicus Therapeutics to evaluate PCs for treating Alzheimer's disease

The Alzheimer's Drug Discovery Foundation announced today that it has awarded a grant of $210,300 to Amicus Therapeutics (Cranbury, NJ) to evaluate small-molecule, orally delivered pharmacological chaperone compounds as a novel approach for treating Alzheimer's disease.

7 May 2010

New method helps study interactions needed for formation of lipopolysaccharides

Scientists have combined chemistry and biology research techniques to explain how certain bacteria grow structures on their surfaces that allow them to simultaneously cause illness and protect themselves from the body's defenses.

26 Apr 2010

New approach may circumvent lost response in insulin due to obesity

Researchers at Children's Hospital Boston have identified a new strategy for treating type 2 diabetes, identifying a cellular pathway that fails when people become obese. By activating this pathway artificially, they were able to normalize blood glucose levels in severely obese and diabetic mice. Their findings will be published online by Nature Medicine on March 28.

29 Mar 2010

Anesthesia can increase behavioral changes, risk of developing Alzheimer's disease

The use of repetitive anesthesia with isoflurane (one of the most common anesthetics by inhalation) increases the risk of developing changes similar to those observed in AD brains in mice with mutations of the amyloid precursor protein (APP). This is the main conclusion of Spanish researchers coordinated by Doctors Maria -ngeles Mena and Justo Garc-a de Y-benes, from CIBERNED (Centro de Investigaci-n Biom-dica en Red de Enfermedades Neurodegenerativas).

25 Mar 2010

Amicus Therapeutics presents additional data from Amigal Phase 2 extension study for Fabry disease

Amicus Therapeutics announced today additional positive preliminary data from its ongoing Phase 2 extension study of its investigational drug Amigal™ (migalastat HCl) for Fabry disease at the Lysosomal Disease Network WORLD Symposium in Miami, Florida.

11 Feb 2010

Anti-inflammatory plant derivative could play a role in cancer treatment, say MCG researchers

Medical College of Georgia researchers are seeking to refine cancer treatment with an anti-inflammatory plant derivative long used in Chinese medicine.

4 Feb 2010

Research offers new hope for rare metabolic disorder

A research team led by biochemist Scott Garman at the University of Massachusetts Amherst has for the first time determined the mechanism of one of the cell's "recycling" enzymes, human alpha-galactosidase or alpha-GAL, as it breaks down substances in the lysosome, the cell's recycling center.

2 Feb 2010