Regenerative cells for treatment of Duchenne muscular dystrophy

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Recent research conducted by Marinee Chuah and Thierry VandenDriessche (Gene Therapy and Regenerative Medicine) is being featured on the cover of the latest issue of Nature Protocols. They provide a set of instructions for the differentiation of so-called induced pluripotent stem cells (iPS cells) into skeletal muscle cells. Pluripotent cells can develop into a range of specific cell types. Normally, pluripotent stem cells are derived from embryos, but here the researchers were able to induce a pluripotent state in adult cells using.

iPS technology was developed several years ago by Japanese Professor Shinya Yamanaka, for which he was awarded the 2012 Nobel Prize in Medicine. He discovered that adult cells could be reprogrammed to make them pluripotent. As a result, iPS cells can be used as a possible alternative to embryonic stem cells. Embryonic stem cells play a crucial role in many medical applications, but also have a number of practical and ethical concerns associated with their use.

One of the main challenges with the use of iPS cells is the ability to turn them into differentiated cells in an efficient manner. These differentiated cells can then be transplanted in patients that suffer from degenerative diseases.

An international collaboration between the team of Dr. Tedesco at the University College London (UCL) and VUB, has lead to the successful  differentiation of iPS cells from patients suffering from Duchenne muscular dystrophy, into muscle cells. In the future, the procedure for the generation of these muscle cells could potentially be used for the treatment of this muscular disorder

Duchenne muscular dystrophy is a hereditary condition in which muscle cells stop working; leaving patients paralyzed and with a greatly reduced life expectancy. Genetically modified muscle cells differentiated from autologous iPS cells will not be rejected by the immune system, as they are from the patients own tissues, but carry a genetic correction against Duchenne Muscular Dystrophy. Such cells could be used for the development of a treatment against this disease.

While treatment using iPS cell will not be ready for many years, the protocol published in Nature Protocols represents a promising step forward in the development of a treatment for this disease.


Maffioletti, Sara M., et al. “Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells.” Nature protocols 10.7 (2015): 941-958.

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