Melanocytes are essential for skin homeostasis and protection, and their loss or misfunction leads to a wide spectrum of diseases. Cell therapy utilizing autologous melanocytes has been used for years as an adjunct treatment for hypopigmentary disorders such as vitiligo. However, these approaches...

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Institute for Stem cell Therapy and Exploration of Monogenic diseases

In 2023, advancing research on rare and ultra-rare monogenic diseases requires the mobilization of significant human and technological resources. Within I-Stem, we are all mobilized around this common objective.


Between scientists and support functions, 75 people currently work at I-Stem.

Teams at the service of ambitious therapeutic objectives


I-Stem brings together within its research teams the expertise and know-how of researchers, teacher-researchers, engineers and technicians. Retinopathies, genodermatoses, Limb-girdle muscular dystrophies, neuromuscular diseases & Myelinated fibers are part of our main scientific themes.


Clinical trials are a crucial step in the development of treatments for any pathology. I-Stem is the sponsor of several trials and supports others through partnerships.


Excellent research requires state-of-the-art equipment. I-Stem is equipped with technological platforms allowing its research teams to optimize their fundamental, pre-clinical and clinical research projects.


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26 April 2023

Thank you to everyone: donors, volunteers mobilized throughout France and abroad, partners, personalities! Thanks to your unfailing mobilization on December 2 and 3, the Telethon 2022 raised 90,839,067 euros! This tremendous result - the best since 2016 - shows how much the French people are in solidarity with the struggle of families.


09 May 2023

Mitochondrial dysfunction involving mitochondria-associated ER membrane (MAM) dysregulation is implicated in the pathogenesis of late-onset neurodegenerative diseases, but understanding is limited for rare early-onset conditions. Loss of the MAM-resident protein WFS1 causes Wolfram syndrome (WS),...

CRISPR/Cas9 editing systems are currently used to generate mutations in a particular gene to mimic a genetic disorder in vitro. Such "disease in a dish" models based on human pluripotent stem cells (hPSCs) offer the opportunity to have access to virtually all cell types of the human body. However...

Glycogen storage disease type III (GSDIII) is a rare genetic disease caused by mutations in the gene encoding the glycogen debranching enzyme (GDE). The deficiency of this enzyme, involved in cytosolic glycogen degradation, leads to pathological glycogen accumulation in liver, skeletal muscles ...

I-Stem is a member of the Institute of Biotherapies for Rare Diseases created by the AFM - Telethon