Grant: Proof of Principle Program Phase I - Drug Development
University of Alberta
$159,196.00
Development of ganglioside-based therapies for Huntington's disease
FY 2010-11
Summary
Huntington's disease (HD) is the most common inherited neurodegenerative disorder and it is characterized by progressive motor dysfunction, cognitive decline and psychiatric symptoms. The disease is caused by a mutation in a protein, huntingtin, that affect the function and survival of brain cells. Currently, there is no cure for this disease. We have recently discovered that the production of gangliosides, lipid molecules that are important for normal functioning of brain cells, is impaired in HD. Our studies will explore the therapeutic application of various gangliosides in HD. We will determine their ability to decrease the toxicity of mutant huntingtin and HD symptoms in animal models. We will also determine the best route of administration of these potential drugs and their distribution in the brain of HD animal models. Our studies will be instrumental to the development and the commercialization of new treatments for HD.
Department
Program
Proof of Principle Program Phase I - Drug Development
Recipient
Location
Alberta, Canada
Recipient Type
Academia
Type
Grant
Source
CIHR Funding Decisions Database
As published
Id
231670
Name
Sipione Simonetta
Region
Alberta
Country
Canada
Orgtype
Institution Paid; Research Institution
Instname2
Neurosciences, Mental Health and Addiction
Theme2
Biomedical
Prcname2
Proof of Principle
Projecttitle
Development of ganglioside-based therapies for Huntington's disease
Programtype2
Operating Grants
Orgnameinp2
University of Alberta
Cihramount2
$159,196
Cihrequipment2
$0
Programname2
Proof of Principle Program Phase I - Drug Development
Keyworddelim
Drug Administration; Ganglioside Metabolism; Huntington'S Disease; Neurodegenerative Disorders
Abstract
Huntington's disease (HD) is the most common inherited neurodegenerative disorder and it is characterized by progressive motor dysfunction, cognitive decline and psychiatric symptoms. The disease is caused by a mutation in a protein, huntingtin, that affect the function and survival of brain cells. Currently, there is no cure for this disease. We have recently discovered that the production of gangliosides, lipid molecules that are important for normal functioning of brain cells, is impaired in HD. Our studies will explore the therapeutic application of various gangliosides in HD. We will determine their ability to decrease the toxicity of mutant huntingtin and HD symptoms in animal models. We will also determine the best route of administration of these potential drugs and their distribution in the brain of HD animal models. Our studies will be instrumental to the development and the commercialization of new treatments for HD.
Competitiondate
201102
Approvedterm2
1 yr 0 mth
Pinamesdelim
Sipione, Simonetta
Approvedterm
1 an 0 mois
Cihrcontribution2
~ $159,196~ $0~
