Therapeutic potential of Synthetic Chemically Modified mRNA to ameliorate cardiac dysfunction associated with Danon disease caused by monogenic LAMP2 mutation.
Grant Data
Project Title
Therapeutic potential of Synthetic Chemically Modified mRNA to ameliorate cardiac dysfunction associated with Danon disease caused by monogenic LAMP2 mutation.
Principal Investigator
Dr Ng, Kwong Man
(Principal Investigator (PI))
Co-Investigator(s)
Professor LUI Kathy Oi Lan
(Co-Investigator)
Professor Tse Hung Fat
(Co-Investigator)
Duration
36
Start Date
2020-10-01
Amount
996285
Conference Title
Therapeutic potential of Synthetic Chemically Modified mRNA to ameliorate cardiac dysfunction associated with Danon disease caused by monogenic LAMP2 mutation.
Presentation Title
Keywords
Autophagic cardiomyopathy, Danon Disease, iPSCs- and Animal-based model, LAMP2 mutation, Modified RNA
Discipline
Cardiovascular Research,Drug Discovery and Delivery
Panel
Biology and Medicine (M)
HKU Project Code
17111320
Grant Type
General Research Fund (GRF)
Funding Year
2020
Status
On-going
Objectives
1 To evaluate the efficiency of modRNA in restoring LAMP2 protein production in cardiomyocytes differentiated from Danon disease patients carrying different LAMP2 mutations. 2 To identify the LAMP2-modRNAs that are effective in ameliorating the cardiac dysfunction in associated with LAMP2 deficiency. 3 To establish a clinically-relevant animal model to verify the therapeutic effects of LAMP2-modRNA.