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Institut des Cellules souches pour le traitement et l'étude des maladies monogéniques (I-STEM)

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
Generating Functional and Highly Proliferative Melanocytes Derived from Human Pluripotent Stem Cells: A Promising Tool for Biotherapeutic Approaches to Treat Skin Pigmentation Disorders Manoubia Saidani, Annabelle Darle, Margot Jarrige, Hélène Polvèche, Lina El Kassar, Nathalie Holic, Cécile Martinat, Christine Baldeschi, Jennifer Allouche 04/01/23 International Journal of Molecular Sciences
Epigenetic regulation of plastin 3 expression by the macrosatellite DXZ4 and the transcriptional regulator CHD4 Julien Come, Cécile Martinat 03/02/23 American Journal of Human Genetics
Photoreceptor Cell Replacement Using Pluripotent Stem Cells: Current Knowledge and Remaining Questions Christelle Monville, Karim Ben M’barek 02/01/23 Cold Spring Harbor perspectives in medicine
MBNL-dependent impaired development within the neuromuscular system in myotonic dystrophy type 1 Julie Tahraoui-Bories, Antoine Mérien, Celine Leteur, Hélène Polvèche, Alexandre Carteron, Jérome Polentes, Margot Jarrige, Cécile Martinat 02/01/23 Neuropathology and Applied Neurobiology
Pluripotent Stem Cells in Disease Modeling and Drug Discovery for Myotonic Dystrophy Type 1 Noémie Bérenger-Currias, Cécile Martinat, Sandrine Baghdoyan 02/01/23 Cells
Propagation of Distinct α-Synuclein Strains Within Human Reconstructed Neuronal Network and Associated Neuronal Dysfunctions Luc Bousset, Ronald Melki, Anselme L. Perrier 01/01/23 Methods in molecular biology (Clifton, N.J.)
Myotonic dystrophy RNA toxicity alters morphology, adhesion and migration of mouse and human astrocytes Hélène Polvèche, Julie Tahraoui-Boris, Cécile Martinat 12/01/22 Nature Communications
Author Correction: Myotonic dystrophy RNA toxicity alters morphology, adhesion and migration of mouse and human astrocytes (Nature Communications, (2022), 13, 1, (3841), 10.1038/s41467-022-31594-9) Hélène Polvèche, Julie Tahraoui-Bories, Cécile Martinat 12/01/22 Nature Communications
Sorting and Manipulation of Human PGC-LC Using PDPN and Hanging Drop Cultures Christelle Monville 12/01/22 Cells
Severe congenital myasthenic syndromes caused by agrin mutations affecting secretion by motoneurons Florine Roussange, Cécile Martinat 10/01/22 Acta Neuropathologica
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 10/01/22 Journal of Investigative Dermatology
Physiology of PNS axons relies on glycolytic metabolism in myelinating Schwann cells Nicolas Tricaud 10/01/22 PLoS ONE
RNA helicase-dependent gene looping impacts messenger RNA processing Hélène Polvèche 09/09/22 Nucleic Acids Research
Mapping the N-Terminal Hexokinase-I Binding Site onto Voltage-Dependent Anion Channel-1 To Block Peripheral Nerve Demyelination Nicolas Tricaud 09/08/22 Journal of Medicinal Chemistry
Human Cytomegalovirus Modifies Placental Small Extracellular Vesicle Composition to Enhance Infection of Fetal Neural Cells In Vitro Alexandra Benchoua 09/01/22 Viruses
Loss of full-length dystrophin expression results in major cell-autonomous abnormalities in proliferating myoblasts Virginie Mournetas, Christian Pinset 09/01/22 eLife
Moxifloxacin rescues SMA phenotypes in patient-derived cells and animal model Camille Januel, Cécile Martinat 08/01/22 Cellular and Molecular Life Sciences
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

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)