ATGC Inc. — Department of Health and Human Services STTR Phase I: OD

Description

Abstract To evaluate the therapeutic applications of stem cells it is essential to develop translational research using several animal species Rabbit is a useful model for biomedical research It has been used to study a number of human diseases Compared to mice rabbits have a more similar embryology and stem cell biology to those of humans an adult body size comparable to human infants that allows for practical surgical procedures and a longer lifespan to permit a clinically relevant research time It will be beneficial to establish rabbit pluripotent stem cell PSC lines as a non murine model system for better understanding human stem cells and for testing the safety and efficacy of new stem cell based technologies The availability of ground state rabbit ESCs in the age of gene editing is expected to create many novel research tools for translational research community Pluripotent rabbit embryonic stem cells rbESCs and induced pluripotent stem cells iPSCs have been reported However these cells share typical properties of primed stem cells and all failed to transmit to the germ cells The ground state or germline transmitting ESCs and iPSCs are only reported in mice and rats It remains to be tested whether ground state ESCs exist in non rodent species The PI of this project made the breakthrough in establishing the first non murine ground state ESCs in rats Here we propose to derive ground state rbESCs In Aim we will establish an Oct PE GFP reporter cell line which would express green florescent in ground state but not in prime state In Aim we will screen small molecule library to identify compounds that are beneficial for the ground state in rabbit ESCs In Aim we will derive new ground state rabbit ESCs Project Narrative In the present work we propose to derive ground state rabbit embryonic stem cells rbESCs To date mouse and rat remain the only mammalian species in which germline transmitting ESCs are available The availability of ground state rabbit ESCs is expected to create many novel research tools for translational research community