16

MAY

24

Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver.

Glycogen storage disease type III (GSDIII) is a rare metabolic disorder due to glycogen debranching enzyme (GDE) deficiency. Reduced GDE activity leads to pathological glycogen accumulation responsible for impaired hepatic metabolism and muscle weakness. To date, there is no curative treatment fo...

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Institute for Stem cell Therapy and Exploration of Monogenic diseases

In 2024, 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.

80

Between scientists and support functions, 80 people currently work at I-Stem.

Teams at the service of ambitious therapeutic objectives

Reasearch
teams

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.

Triails
clinics

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.

stemCARE
Technology plateform

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.

News

  • News
  • Publications
Event

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.

NGS.

21 May 2024

Transcription termination fine tunes gene expression and contributes to specify the function of RNAs in eukaryotic cells. Transcription termination of hepatitis B virus (HBV) is subjected to the recognition of the canonical polyadenylation signal (cPAS) common to all viral transcripts. The regula...

LGMDs.NGS.

16 May 2024

Glycogen storage disease type III (GSDIII) is a rare metabolic disorder due to glycogen debranching enzyme (GDE) deficiency. Reduced GDE activity leads to pathological glycogen accumulation responsible for impaired hepatic metabolism and muscle weakness. To date, there is no curative treatment fo...

Retinopathies.

16 April 2024

The retinal pigment epithelium (RPE), a multifunctional cell monolayer located at the back of the eye, plays a crucial role in the survival and homeostasis of photoreceptors. Dysfunction or death of RPE cells leads to retinal degeneration and subsequent vision loss, such as in Age-related macular...

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