Title：Towards Therapies Combining Reprogramming and Genetic Engineering
Date：Friday, June 26^th 2020
Time：13:00 –13:30 EDT

Abstract：Reprogramming holds the promise to generate autologous cells for cell transplantation-based tissue repair. In addition to simply derived and regenerate lost cells, the advent of facile genetic engineering allows now to not only repair genetic defects causing disease ex vivo for cell transplantation but also the engineering of disease interfering systems to bestow transplanted cells with therapeutic interventions.
As a first step, we have been focusing on rare monogenetic diseases that have a solid scientific basis for a beneficial cell transplantation. We have begun with the rare skin disease Epidermolysis Bullosa to implement induced pluripotent stem (iPS) and efficient Cas9-based genetic engineering. Manufacturing process optimized in the laboratory need to transition into good manufacturing practice (GMP) and all reagents need to be certified or exchanged to certifiable reagents to satisfy FDA-regulations of conducting Phase 1 clinical trials.

Speaker：Marius Wernig, M.D., Ph.D. Institute for Stem Cell Biology and Regenerative Medicine, Stanford University

 
Visit our booth and posters！　　Booth : Ajinomoto Co., Inc.　
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