Viral-Vector Integration-Free Human Induced Pluripotent Stem Cells Platform Derived from Urine Samples for Disease Diagnosis and Drug Screening

Professor Tse, Hung Fat   (Project Coordinator (PC))

Dr Lai Kevin Wing Hon   (Co-Investigator)

Professor Esteban Miguel   (Co-Investigator)

Dr Ho Jenny Chung Yee   (Co-Investigator)

18

2013-07-01

993031

Viral-Vector Integration-Free Human Induced Pluripotent Stem Cells Platform Derived from Urine Samples for Disease Diagnosis and Drug Screening

Pluripotent Stem Cells, Viral-Vector Integration-Free

Others - Medicine, Dentistry and Health

ITS/303/12

Innovation and Technology Support Programme (Tier 3)

2012

Completed

Objective-1: Generation of urine human iPSC [hiPSC](0-6 months): - Establish a protocol to generate hiPCS from urine epithelial cells in patients with different inherited human diseases, such as familial hypercholesterolemia and dilated cardiomyopathy using different combinations of episomal vectors and small molecules to improve the efficiency of reprogramming. - Characterize the pluripotent status of hiPSC-derived from urine samples and compare their reprogramming and differentiation efficiency with those hiPSCs generated from conventional approach using skin fibroblast generated from the same donor. Objective-2: Differentiation of hiPSC-derived cardiomyocytes and hepatocytes (7-12 months): - Establish a platform for large scale suspension culture (up to 5-liters) using bioreactor to yield sufficient number (~100 million) of urine hiPSC-derived cardiomyocytes for modeling dilated cardiomyopathy and performing drug screening. - Enhance the efficiency by refining our existing protocol for differentiation of P. 11 hepatocytes from urine hiPSC using different small molecules and growth factors to provide 10x96-well plates for modeling familial hypercholesterolemia and performing drug screening. Objective-3: Disease modeling and drug screening (13-18 months):