2023-10-20 09:03:17
Described for the first time in 2006 by Japanese researcher Shinya Yamanaka, which earned him the Nobel Prize in Medicine in 2012, iPS induced pluripotent stem cells (for induced pluripotent stem cells) are increasingly used in the laboratory.
Did you say iPS induced pluripotent stem cells?
“Stem cells” are undifferentiated cells capable of self-renewal and differentiation in order to give rise to any specialized cells in our body (blood cells, muscle cells, neurons, etc.).
Unlike embryonic stem cells which come from a surplus embryo not used following fertilization in vitro, iPS cells come from cells taken from an adult which have been “rejuvenated” to restore their ability to transform into any type of cell. The advantage? In addition to overcoming the ethical problems linked to embryo manipulation, these cells can also be used to model diseases, such as neuromuscular diseases, provided that the adult cells used initially are diseased.
Reproduce complex systems like the neuromuscular junction
This tool can thus prove useful for modeling the neuromuscular junction, the contact zone between the motor neuron and its muscular target, which plays a major role in many neuromuscular diseases. It will make it possible to study each component individually but also the interactions between motor neurons and muscles, synaptic vesicles, etc.
Very recently, it has been shown that iPS cells can also be used to develop small organs called “organoids”. These small three-dimensional structures resemble the entire organ, making it possible to understand its development and functioning, or to test potential treatments.
In numbers
28 days to allow iPS cells to differentiate into motor neurons.
3 months to obtain a 3D neuromuscular junction.
From laboratory to clinical trial
Researchers from the I-Stem institute have thus succeeded, thanks to high-throughput screening, in identifying metformin, an antidiabetic drug already on the market, as an effective treatment in stem cells from people suffering from Steinert’s disease. This was followed by a phase II trial which involved 40 participants and showed, following one year of treatment, that patients treated with metformin (versus placebo) gained motor skills and found a more stable gait. A new trial in Steinert’s disease is being prepared in France (phase III).
Two other drug candidates, bosutinib and retigabine, also showed their effectiveness on iPS cells from patients with Charcot disease (ALS) before being evaluated in clinical trials.
1697794449
#Human #stem #cells #induced #pluripotency #valuable #tool #understand #treat #neuromuscular #diseases
