Programmable RNA Pseudouridylation for Premature Termination Codon Suppression as Gene Therapy for Cardiac Laminopathy

View Statistics Email Alert RSS Feed

Grant Data
Project Title
Programmable RNA Pseudouridylation for Premature Termination Codon Suppression as Gene Therapy for Cardiac Laminopathy
Principal Investigator
Professor Tse, Hung Fat   (Project Coordinator (PC))
Co-Investigator(s)
Professor Wong Chun Ka   (Co-principal investigator)
Yi Chengqi   (Co-principal investigator)
Duration
12
Start Date
2024-06-30
Amount
2661362
Conference Title
Programmable RNA Pseudouridylation for Premature Termination Codon Suppression as Gene Therapy for Cardiac Laminopathy
Keywords
1) Dilated cardiomyopathy 2) Cardiac laminopathy 3) Lamin A/C 4) Gene therapy 5) RNA therapeutics
Discipline
Cardiovascular ResearchGenomic Medicine
Panel
Biology and Medicine (M)
HKU Project Code
C7096-23G
Grant Type
Collaborative Research Fund (CRF) - Group Research Project 2023/2024
Funding Year
2024
Status
On-going
Objectives
1. To assess the safety and efficacy of ""RESTART"" system for achieving prematuretermination codon (PTC) readthrough in-vitro in patient-specific human induced pluripotentstem cells (hiPSCs) derived cardiomyocytes (hiPSC-CMs) generated from dilatedcardiomyopathy (DCM) patients harboring LMNA mutations.2. To determine the most effective method for in-vivo transfection of ""RESTART"" system invivo in LMNAR225X/WT mouse model of DCM.3. To assess the in-vivo safety and efficacy of ""RESTART"" system for achieving PTCreadthrough in LMNAR225X/WT mouse model of DCM.4. To determine the therapeutic efficacy of ""RESTART"" system in attenuation of the DCMphenotypes in-vitro hiPSC-CMs; and in-vivo LMNAR225X/WT mouse model of DCM.