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

Présentation du laboratoire

L'Institut des cellules Souches pour le Traitement et l'Etude des maladies Monogéniques (I-Stem) est un centre de R&D dédié à l'élaboration de traitements fondés sur les potentiels offerts par les cellules souches pluripotentes et applicable aux maladies rares d'origine génétique. Son domaine d'activité s'étend depuis la recherche fondamentale de mécanismes pathologiques jusqu'au transfert de nouvelles thérapies à la recherche clinique. Les grandes indications pathologiques étudiées actuellement sont relatives aux maladies du muscle, des motoneurones et de la peau.

Les 20 dernières publications

Titre Auteurs Date de publication Source
A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III Lucille Rossiaud, Xavier Nissan, Lucile Hoch 16/01/2024 Journal of Clinical Investigation
Nucleolar reorganization after cellular stress is orchestrated by SMN shuttling between nuclear compartments Cécile Martinat 01/12/2023 Nature Communications
Ghrelin delays premature aging in Hutchinson-Gilford progeria syndrome Xavier Nissan 01/12/2023 Aging Cell
Semi-automated optimized method to isolate CRISPR/Cas9 edited human pluripotent stem cell clones Elie Frank, Michel Cailleret, Pascal Fragner, Jérôme Polentes, Elise Herardot, Lina El Kassar, Karine Giraud-Triboult, Christelle Monville, Karim Ben M’Barek 01/12/2023 Stem Cell Research and Therapy
Molecular Analysis of a Congenital Myasthenic Syndrome Due to a Pathogenic Variant Affecting the C-Terminus of ColQ Laurine Merriadec, Florine Roussange, Cécile Martinat 01/11/2023 International Journal of Molecular Sciences
Establishment of heterozygous and homozygous SHANK3 knockout clonal pluripotent stem cells from the parental hESC line SA001 using CRISPR/Cas9 Laure Chatrousse, Thifaine Poullion, Lina El Kassar, Karine Giraud-Triboult, Claire Boissart, Alexandra Benchoua 01/10/2023 Stem Cell Research
Generation of three induced pluripotent stem cell lines from patients with glycogen storage disease type III Lucille Rossiaud, Emilie Pellier, Manon Benabides, Xavier Nissan, Lucile Hoch 01/10/2023 Stem Cell Research
Unlocking the Complexity of Neuromuscular Diseases: Insights from Human Pluripotent Stem Cell-Derived Neuromuscular Junctions Morgan Gazzola, Cécile Martinat 01/10/2023 International Journal of Molecular Sciences
CRISPR/Cas9-mediated generation of human embryonic stem cell sub-lines with HPRT1 gene knockout to model Lesch Nyhan disease Claire Boissart, Laure Chatrousse, Thifaine Poullion, Lina El Kassar, Karine Giraud-Triboult, Alexandra Benchoua 01/09/2023 Stem Cell Research
Depletion of WFS1 compromises mitochondrial function in hiPSC-derived neuronal models of Wolfram syndrome Axel Sciauvaud, Sandra Pourtoy-Brasselet, Margot Jarrige, Hélène Polvèche, Laetitia Aubry 09/05/2023 Stem Cell Reports
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 01/04/2023 International Journal of Molecular Sciences
Epigenetic regulation of plastin 3 expression by the macrosatellite DXZ4 and the transcriptional regulator CHD4 Julien Come, Cécile Martinat 02/03/2023 American Journal of Human Genetics
Photoreceptor Cell Replacement Using Pluripotent Stem Cells: Current Knowledge and Remaining Questions Christelle Monville, Karim Ben M’Barek 01/02/2023 Cold Spring Harbor perspectives in medicine
MBNL-dependent impaired development within the neuromuscular system in myotonic dystrophy type 1 Julie Tahraoui-Bories, Antoine Mérien, Céline Leteur, Hélène Polvèche, Alexandre Carteron, Jérôme Polentes, Margot Jarrige, Cécile Martinat 01/02/2023 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 01/02/2023 Cells
Propagation of Distinct α-Synuclein Strains Within Human Reconstructed Neuronal Network and Associated Neuronal Dysfunctions Simona Gribaudo, Anselme L. Perrier 01/01/2023 Methods in molecular biology (Clifton, N.J.)
SplicingLore: A web resource for studying the regulation of cassette exons by human splicing factors Hélène Polvèche 01/01/2023 Database : the journal of biological databases and curation
Identification of signaling pathways modifying human dopaminergic neuron development using a pluripotent stem cell-based high-throughput screening automated system: purinergic pathways as a proof-of-principle Claire Boissart, Marie Lasbareilles, Johana Tournois, Laure Chatrousse, Thifaine Poullion, Alexandra Benchoua 01/01/2023 Frontiers in Pharmacology
Myotonic dystrophy RNA toxicity alters morphology, adhesion and migration of mouse and human astrocytes Hélène Polvèche, Julie Tahraoui-Boris, Cécile Martinat 01/12/2022 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 01/12/2022 Nature Communications

Nombre de publications du laboratoire par domaine scientifique (2016-2021)

Chaque publication du laboratoire peut être rangée dans une ou plusieurs disciplines scientifiques : la figure ci-dessus présente le nombre de publications du laboratoire pour chaque discipline de la classification ASJC (Elsevier)