Regenerative Medicine - Stem Cells
Understanding functional significance of gene mutations using human iPSC cells
Human pluripotent stem cells has ushered in a new era of biomedical research and applied medicine by harnessing the power of somatic cell reprogramming. This reprogramming is accomplished by the delivery of the genes OCT4, SOX2, c-MYC and KLF-4 leading to the de-differentiation of the cells into a pluripotent stem cell state as described by Yamanaka et al29. This technology holds the potential to generate patient specific cell and tissue types that can be used for patient-specific, personalised therapies. Importantly, iPSCs provide an opportunity to non-invasively study the disease specific cell types from actual patients in the laboratory without having to go through the lengthy and expensive process of generating transgenic or mutant animal models. The cellular molecular processes driving the disease in patients can be recapitulated easily and targeted for genetic manipulation and correction, paving the way for ex-vivo therapy. To this end, a number of methods have been developed successfully to redifferentiate the patient derived iPSC cells to retinal/corneal cell types. These cells can therefore also be used for investigating molecular functions of the mutated proteins and aberrant cellular pathways as well as testing gene or drug based therapies for treatment of disorders like RP.
Induced Pluripotent Stem cells (iPSCs)
Patient derived iPSCs hold immense potential in understanding disease etiology and towards developing personalized medicine. Here at GROW lab, we are developing iPSCs to model ocular disorders to obtain insight into the disease mechanisms and develop potential drug screening tools