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Laboratory

I-Stem – Institute for Stem cell Therapy and Exploration of Monogenic diseases


I-STEM INSERM/UEVE UMR 861 CRCT, 28 rue Henri Desbruères
91100 CORBEIL-ESSONNES
Phone : +33 1 69 90 85 17
Email : sfacchinato@istem.fr

Manager : MARTINAT Cécile
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#Genomics / Postgenomics

  • Cell therapy
  • Disease modeling
  • Pharmacology
  • Stem cells
  • Monogenic diseases

Supervisors:

AFM TéléthonUEVE - Tutelle laboratoire génopolitainInserm - tutelle de laboratoires génopolitains

Field of Activity


Postgenomics and biotherapies of monogenic diseases.

I-STEM can be conceptualized with three key terms: “stem cells”, “therapeutics” and “monogenic diseases”. The institute evaluates the therapeutic potential of all types of stem cells in the setting of monogenic diseases. Within this mission, I-STEM explores more particularly substitutive cellular therapies for degenerative diseases on one hand and the use of pathological stem cell lines as targets for the screening of potentially therapeutic compounds on the other.

The teams at I-Stem develop research programs employing embryonic stem cells and induced pluripotent stem cells with the aim of creating therapeutics for rare genetic diseases.

Three major research orientations are forwarded in parallel:

  • Regenerative medicine involves the production and differentiation of pluripotent stem cells, thereafter implanted in patients to regenerate damaged tissues.
    Three programs are underway.
    – The first, a collaborative effort with the AP-HP and currently under review at ANSM, looks to develop cutaneous grafts to treat sickle-cell anemia-associated skin ulcers.
    – The second is being carried out in partnership with teams from the Institut de la Vision with the objective of treating retinitis pigmentosa through the implantation of retinal pigment epithelial cells.
    – And the third, in collaboration with MirCen, is targeting the implantation of intracerebral nerve cells to treat Huntington’s disease.
  • Pathological modeling involves the study of cells derived from pluripotent stem cell lines sourced from diseased patients or embryos.
    I-Stem focuses on pathologies affecting muscle (type 1 myotonic dystrophy, Duchenne muscular dystrophy) the central nervous system (infantile spinal muscular atrophy, genetic autism, Lesch–Nyhan syndrome, Wolfram syndrome) or other tissues (neurofibromatosis, familial adenomatous polyposis, progeria).
  •  Identification of pharmacological compounds with potential for development as treatments for these pathologies.
    This branch of research, performed with the support of major technological platforms that enable high volume cell production and medium and high throughput drug screening, has already led to the identification of numerous compounds having the potential to counter pathological mechanisms. Work done on cells donated by patients with type 1 myotonic dystrophy, Huntington’s disease, Lesch–Nyhan syndrome or progeria has already been fruitful. A clinical trial to evaluate the therapeutic efficacy of a compound identified in the setting of type 1 myotonic dystrophy is underway, in collaboration with the AP-HP.
  • TEAMS

    Maladies neurodégénératives (Maladie de Huntington) dirigé par Anselme PERRIER

    Maladies du motoneurone (Maladie de Steinert) dirigé par Cécile MARTINAT

    Myopathie de Duchenne dirigé par Christelle MONVILLE

    Génodermatoses dirigé par Christine BALDESCHI

    Biothérapie des lésions neuro-vasculaires dirigé par Brigitte ONTENIENTE

     

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