March 17, 2014
A research group led by Francesco Saverio Tedesco, M.D., Ph.D., will assess whether the iPS cells will enable production of large quantities of progenitor cells that can be transplanted into a patient’s muscle following genetic correction, or as part of drug development platforms.
Dr. Tedesco’s team will also investigate the potential for treating muscular dystrophy through new gene and cell therapy strategies using artificial human chromosomes and novel biomaterials.
The research will be funded with $250,000 from Takeda’s New Frontier Sciences group, designed to support research that could eventually lead to drug discovery and development. “With Takeda’s backing, my team and I will be building on the processes we’ve already shown to be promising and which could pave the way for the development of novel strategies for both cell therapies and drug discovery in muscle disorders,” Dr. Tedesco said in a statement.
Through previous work using a mouse model of Duchenne muscular dystrophy, Dr. Tedesco and colleagues in 2011 showed the potential of preclinical gene replacement therapy using an artificial human chromosome. A year later in a separate study, Dr. Tedesco and his team demonstrated the potential of genetically corrected iPS cells transplanted into another mouse model of a genetic muscle disorder, limb-girdle muscular dystrophy 2D.
“That several different strands of their research have already borne fruit was strong evidence for us of the translational potential of Dr Tedesco’s research for muscular dystrophies,” added Gordon Wong, D.Phil., Head of New Frontier Science at Takeda.
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