In a study published today in the journal EMBO Molecular Medicine1, the team led by Prof. Nicolas Lévy identifies the mechanism associated with the accumulation of progerin, a toxic protein produced in the course of ageing, and demonstrates the therapeutic potential of a new drug – MG132 – to treat progeria, a rare syndrome involving premature and accelerated ageing. Nicolas Lévy and his team have demonstrated the ability of...
Cholesterol plays a central role in many living processes. In a new study, a team led by Catherine-Laure Tomasetto, Inserm research director at the Institute of Genetics and Molecular and Cellular Biology (Inserm/CNRS/Université de Strasbourg) reveals the role played by the STARD3 protein in the distribution of cholesterol within cells. A little like molecular velcro, this protein has the capacity to form membrane contacts between two cell organelles, enabling...
An international collaborative study coordinated by Frédéric Laumonnier (Unit 930 “Imaging and Brain” Inserm/University of Tours) and Yann Hérault of the Institute of Genetics and Molecular and Cellular Biology (Inserm/ CNRS/ University of Strasbourg) provides new and original findings on the pathophysiological role of the contact areas between neurons in certain brain disorders. The study reveals that mutation of one of the genes involved in intellectual disability and autism...
A team of researchers in France, led by Dr. Ana Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, achieved a new step towards the treatment of myotubular myopathy by gene therapy. The researchers demonstrated the efficacy of administration of a therapeutic vector by a single intravenous injection and identified the dose that restores long-term muscular strength in a large animal...
French researchers have identified a marker that makes it possible to differentiate “dormant” HIV-infected cells from healthy cells. This discovery will make it possible to isolate and analyze reservoir cells which, by silently hosting the virus, are responsible for its persistence even among patients receiving antiviral treatment, whose viral load is undetectable. It offers new therapeutic strategies for targeting infected cells. This research is part of the ANRS strategic...
Some cancer cells are resistant to treatment and persist. If they are capable of proliferating again, even a very small number of these cells may be enough to reconstitute a tumour after or despite treatment. Various approaches to eliminate these “cancer stem cells” (CSCs) have been tried in recent years: targeted therapies, vaccination and tumour starvation. In an article published in the journal Cell Reports, Christophe Ginestier, Inserm Research...
A team led by Prof. Marina Cavazzana, working at Necker Hospital for Sick Children, AP-HP, and the Imagine Institute (AP-HP/Inserm/Paris Descartes University) performed gene therapy on a 13-year-old patient with severe sickle cell anaemia in October 2014 as part of a phase I/II clinical trial. Conducted in collaboration with Prof. Philippe Leboulch (CEA/Faculties of Medicine at Paris-Sud and Harvard Universities), who developed the vector used, and supervised the preclinical...
A national trial on membranous glomerulonephritis in 80 patients was coordinated by Prof. Pierre Ronco, of the Department of Nephrology and Dialysis at Tenon Hospital AP-HP, of the Inserm Unit “Rare and common kidney diseases, matrix remodelling and tissue repair” [1] and Pierre and Marie Curie University, and by Dr Karine Dahan, of the Nephrology Day Hospital at Tenon Hospital AP-HP. This serious autoimmune disease is the most common...
On 13 June last, the Inserm Ethics Committee assembled over a hundred individuals at its annual seminar. All those present had the benefit of an ethical perspective on many problems posed by biomedical research. One of the questions addressed was that of CRISPR-Cas9 technology. The Ethics Committee has devoted a specific opinion to it, while the NIH has just obtained a first green light for a human cancer immunotherapy...
An international team, including researchers in France at Inserm, CNRS and the University of Strasbourg, brought together at IGBMC[1] is lifting the veil on the molecular mechanisms causing heart dysfunctions in myotonic dystrophy, a genetic disease affecting one person in 8,000. This new study, published this week in Nature Communications, could contribute to discovering a treatment.