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Japan-based Takeda Pharmaceutical and University College London (UCL) will work together for conducting research on tackling muscle disorders, with a particular focus on muscular dystrophy.

The research group of Dr Francesco Saverio Tedesco from the UCL department of Cell and Developmental Biology will conduct the research.

Dr Tedesco’s team will focus on a study of muscular regeneration and the potential for stem cell therapies to treat muscular dystrophy, in particular induced pluripotent (iPS) stem cells.

In addition, Dr Tedesco’s team will also study the potential for treating muscular dystrophy through developing novel gene and cell therapy strategies using artificial human chromosomes and novel biomaterials.

Using this approach, Dr Tedesco hopes to overcome a number of current limitations to develop effective treatments for muscular dystrophies.

Through the use of the modified stem cells, the team hopes to produce large quantities of progenitor cells that can be later transplanted into a patient’s muscle following genetic correction or to be used for drug development platforms.

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Dr Tedesco’s team will attempt to produce the modified stem cells, which can be applied more easily in a clinical context, to reduce obstacles that might limit their possible future use in clinical studies.

Through previous work using a mouse model of Duchenne muscular dystrophy, the team has demonstrated the potential of pre-clinical gene replacement therapy using an artificial human chromosome.

"This funding from Takeda comes at a crucial time for building on the team’s research to develop new approaches for potentially life-changing treatments for muscular dystrophies."

In a separate study, Dr Tedesco’s team demonstrated the potential of genetically corrected iPS cells, which had been transplanted into another mouse model of a genetic muscle disorder (limb-girdle muscular dystrophy 2D).

Dr Tedesco said this funding from Takeda comes at a crucial time for building on the team’s research to develop new approaches for potentially life-changing treatments for muscular dystrophies.

"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.

Takeda’s New Frontier Sciences group is supporting the research through funding of $250,000.

The New Frontier Sciences group aims to support research that could eventually lead to drug discovery and development.


Image: Takeda Midosuji Building in Osaka, Japan. Photo: courtesy of J o.