Generation of dysferlin deficient inducible pluripotent stem cell lines
Project Summary: CDI will employ their MyCell® Products line to create dysferlinopathy patient-derived iPSC lines. All five of the donors for this project have been recruited. iPSC technology is based on reprogramming adult cells to a pluripotent stem cell state, whereby the resulting iPSCs, and the somatic cells derived from them, carry the genotypic backgrounds of the original donors and can be used to model their disease. A primary goal of the Jain Foundation is to use these disease models for drug discovery and assessing therapeutic interventions.