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Optogenetically controlled human functional motor endplate for testing botulinum neurotoxins

The lack of physiologically relevant and predictive cell-based assays is one of the major obstacles for testing and developing botulinum neurotoxins (BoNTs) therapeutics. Human-induced pluripotent stem cells (hiPSCs)-derivatives now offer the opportunity to improve the relevance of cellular models and thus the translational value of preclinical data. The authors investigated the potential of hiPSC-derived motor … [Read more]

Contribution of Human Pluripotent Stem Cell-Based Models to Drug Discovery for Neurological Disorders

  One of the major obstacles to the identification of therapeutic interventions for central nervous system disorders has been the difficulty in studying the step-by-step progression of diseases in neuronal networks that are amenable to drug screening. Recent advances in the field of human pluripotent stem cell (PSC) biology offers the capability to create patient-specific … [Read more]

Human iPSC-derived neurons reveal early developmental alteration of neurite outgrowth in the late-occurring neurodegenerative Wolfram syndrome

  Recent studies indicate that neurodegenerative processes that appear during childhood and adolescence in individuals with Wolfram syndrome (WS) occur in addition to early brain development alteration, which is clinically silent. Underlying pathological mechanisms are still unknown. We have used induced pluripotent stem cell-derived neural cells from individuals affected by WS in order to reveal … [Read more]

SIStemA: A large and standardized collection of transcriptome data sets for human pluripotent stem cell research

  Human pluripotent stem cells have ushered in an exciting new era for disease modeling, drug discovery, and cell therapy development. Continued progress toward realizing the potential of human pluripotent stem cells will be facilitated by robust data sets and complementary resources that are easily accessed and interrogated by the stem cell community. In this … [Read more]

CRISPR gene editing in pluripotent stem cells reveals the function of MBNL proteins during human in vitro myogenesis

    Alternative splicing has emerged as a fundamental mechanism for the spatiotemporal control of development. A better understanding of how this mechanism is regulated has the potential not only to elucidate fundamental biological principles, but also to decipher pathological mechanisms implicated in diseases where normal splicing networks are mis-regulated. Here, the authors took advantage … [Read more]

Hutchinson-Gilford progeria syndrome: Rejuvenating old drugs to fight accelerated ageing

  What if the next generation of successful treatments was hidden in the current pharmacopoeia? Identifying new indications for existing drugs, also called the drug repurposing or drug rediscovery process, is a highly efficient and low-cost strategy. First reported almost a century ago, drug repurposing has emerged as a valuable therapeutic option for diseases that … [Read more]

AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A

  Charcot-Marie-Tooth disease 1 A (CMT1A) results from a duplication of the PMP22 gene in Schwann cells and a deficit of myelination in peripheral nerves. Patients with CMT1A have reduced nerve conduction velocity, muscle wasting, hand and foot deformations and foot drop walking. Here, the authors evaluate the safety and efficacy of recombinant adeno-associated viral … [Read more]

Duchenne myopathy: Myogenesis modelled by human pluripotent stem cells, a multi‐omic study of disease early onset.

  We are proud to share the new publication by Virginie Mournetas and the IStem “Muscle Diseases” team:     Mournetas V, et al (2021). Myogenesis modelled by human pluripotent stem cells: a multi‐omic study of Duchenne myopathy early onset. Journal of Cachexia, Sarcopenia and Muscle. doi : 10.1002/jcsm.12665   We invite you to visit … [Read more]

My thesis in 180s’ 2020 : Élise Hérardot

    Doctoral student in Immunology at IStem, Élise Herardot is interested in the “reconstitution of a retina by bioengineering based on human pluripotent stem cells for the treatment of diseases affecting vision” alongside Karim Ben M’Barek and Christelle Monville. In this context, she took part in the final of Ma Thèse en 180s at … [Read more]

Stem cells: just 10 years from research to first clinical trials

I-Stem research laboratory was celebrating its 10 years in cell therapy R & D last October 27th in Paris: the right time to organize a press conference gathering international top medical opinion leaders in order to make an update on their pioneering work and the major steps forward to innovative stem cell therapies. Cross funded … [Read more]