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Institut des Cellules souches pour le traitement et l'étude des maladies monogéniques

Laboratory presentation

I-Stem is the largest French laboratory for research and development dedicated to human pluripotent stem cells, of embryonic origin or obtained by reprogramming gene. The specific vocation of I-Stem is to explore all the therapeutic potential of human pluripotent stem cells for applications in patients affected by rare diseases of genetic origin. In this context, our teams are developing two major areas of research. The first one is cell therapy, which aims to replace lost or diseased cells to other cells with the same characteristics, produced in the laboratory from pluripotent stem cells. The second area is pharmacology based on automated screening of large libraries of compounds with therapeutic potential, following modeling of molecular mechanisms associated with diseases, as revealed by the study of pluripotent stem cells from affected donors. I-Stem teams are currently working on a dozen genetic diseases that affect different organs. The Institute also hosts every year many researchers interested in other diseases and provides training and technology support.

The 20 latest publications

Title Authors Publication date Source
Dual Blockade of Misfolded Alpha-Sarcoglycan Degradation by Bortezomib and Givinostat Combination Lucile Hoch, Celine Bruge, Manon Benabides, Emilie Pellier, Johana Tournois, Gurvan Mahé, Xavier Nissan 04/27/22 Frontiers in Pharmacology
Muscle cells of sporadic amyotrophic lateral sclerosis patients secrete neurotoxic vesicles Cécile Martinat 04/01/22 Journal of Cachexia, Sarcopenia and Muscle
Generation of a heterozygous SCN5A knockout human induced pluripotent stem cell line by CRISPR/Cas9 edition Lina El Kassar, Karine Giraud-Triboult 04/01/22 Stem Cell Research
Clinical Grade Human Pluripotent Stem Cell-Derived Engineered Skin Substitutes Promote Keratinocytes Wound Closure In Vitro Raif Eren Ayata, Gilles Lemaitre, Christine Baldeschi 04/01/22 Cells
Human-Induced Pluripotent Stem Cell‒Derived Keratinocytes, a Useful Model to Identify and Explore the Pathological Phenotype of Epidermolysis Bullosa Simplex Manon Bonnette, Sabrina Martineau, Nathalie Holic, Gilles Lemaitre, Cécile Martinat, Christine Baldeschi 01/01/22 Journal of Investigative Dermatology
AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A Patrick Aubourg 12/01/21 Nature Communications
Optogenetically controlled human functional motor endplate for testing botulinum neurotoxins Juliette Duchesne de Lamotte, Jérôme Polentes, Florine Roussange, Léa Lesueur, Pauline Feurgard, Anselme L. Perrier, Cécile Martinat 12/01/21 Stem Cell Research and Therapy
Contribution of human pluripotent stem cell-based models to drug discovery for neurological disorders Alexandra Benchoua, Marie Lasbareilles, Johana Tournois 12/01/21 Cells
Human iPSC-derived neurons reveal early developmental alteration of neurite outgrowth in the late-occurring neurodegenerative Wolfram syndrome Sandra Pourtoy-Brasselet, Axel Sciauvaud, Maria Gabriela Boza-Moran, Michel Cailleret, Margot Jarrige, Hélène Polvèche, Jérôme Polentes, Cécile Martinat, Marc Peschanski, Laetitia Aubry 11/04/21 American Journal of Human Genetics
Dual imaging of dendritic spines and mitochondria in vivo reveals hotspots of plasticity and metabolic adaptation to stress Nicolas Tricaud 11/01/21 Neurobiology of Stress
Exosomes: Multi-faceted natural intercellular messengers as new candidates for the treatment of myopathies? Judith Lorant 11/01/21 Medecine/sciences
Modulation of alternative splicing during early infection of human primary B lymphocytes with Epstein-Barr virus (EBV): a novel function for the viral EBNA-LP protein Hélène Polvèche 10/11/21 Nucleic Acids Research
Chimeric embryos and pseudo-embryos: An alternative to human embryos for research Cécile Martinat 08/01/21 Medecine/sciences
Emerging opportunities in human pluripotent stem-cells based assays to explore the diversity of botulinum neurotoxins as future therapeutics Juliette Duchesne de Lamotte, Anselme L. Perrier, Cécile Martinat 07/02/21 International Journal of Molecular Sciences
Hutchinson-Gilford progeria syndrome: Rejuvenating old drugs to fight accelerated ageing Solenn M. Guilbert, Xavier Nissan 06/01/21 Methods
Integrative Cell Type-Specific Multi-Omics Approaches Reveal Impaired Programs of Glial Cell Differentiation in Mouse Culture Models of DM1 Hélène Polvèche 05/05/21 Frontiers in Cellular Neuroscience
Human cytomegalovirus infection is associated with increased expression of the lissencephaly gene PAFAH1B1 encoding LIS1 in neural stem cells and congenitally infected brains Alexandra Benchoua 05/01/21 Journal of Pathology
Increasing brain palmitoylation rescues behavior and neuropathology in Huntington disease mice Morgane Louessard, Patricia Lino, Anselme L. Perrier 03/01/21 Science advances
Dynamic extrinsic pacing of the HOX clock in human axial progenitors controls motor neuron subtype specification Hynek Wichterle 03/01/21 Development (Cambridge)
Myogenesis modelled by human pluripotent stem cells: a multi-omic study of Duchenne myopathy early onset Virginie Mournetas, Emmanuelle Massouridès, Jean Baptiste Dupont, Hélène Polvèche, Margot Jarrige, Alan R.L. Dorval, Christian Pinset 02/01/21 Journal of Cachexia, Sarcopenia and Muscle

Number of publications of the laboratory by scientific field (2016-2021)

Every paper can be classified in one or more scientific fields. The figure below shows the lab's number of publications in each scientific field, according to the ASJC classification (Elsevier)