Head: Anne Bigot
With the development of numerous innovative therapeutic approaches for genetic diseases, targeting DNA, RNA or proteins, muscle cells isolated from patients, known as myoblasts, represent an ideal in vitro model for evaluating these approaches for neuromuscular diseases. These cells have the advantage of being simple to use, reducing the number of animal experiments and also of carrying the patient’s exact mutation in its own genetic environment. However, there are limits to these in vitro approaches: human somatic cells have a limited capacity for proliferation, regulated by the mitotic clock, and reach replicative senescence after a defined number of divisions. This proliferation limit is reached even earlier in degenerative diseases. MyoLine neutralises replicative senescence in human muscle cells using double transduction with lentiviral vectors, creating immortal human cell lines.
Since its creation in 2007, the Institute of Myology’s human cell immortalisation platform has generated nearly 200 human myoblast lines isolated from patients suffering from more than 36 different diseases (DMD, LGMD, FSHD, SMA, etc.). As access to muscle biopsies may be limited for certain diseases, we have also developed the immortalisation of skin fibroblasts. These immortalised fibroblasts are then transduced by an inducible transcription factor MyoD, and these myoconverted cells form myotubes and express muscle markers like muscle cells.
The loss of muscle mass in patients with neuromuscular diseases is often accompanied by its replacement by fibrous and/or adipose tissue. In recent years, research in this field has focused on the role played by fibro-adipogenic progenitors (FAPs) because of their ability to differentiate into adipocytes or fibroblastic cells. To support this research, MyoLine is currently developing models of FAPs isolated from various muscular dystrophies.
For several years, these cell lines have been made available to the scientific community on a collaborative basis, and many international laboratories are already using them. Where consent signed by the donor permits, cell lines can also be used under material transfer agreements for the development of therapeutic tools by private partners.
Please send your requests to anne.bigot@sorbonne-universite.fr & vincent.mouly@sorbonne-universite.fr
Team members
Bensalah Mona, Engineer
Bigot Anne, Research Project Leader, Platform Manager
Diarra Rokiatou, Apprentice
Mamchaoui Kamel, Engineer
Mouly Vincent, Research Director Emeritus
Ohana Jessica, Engineer
