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      DREAMS - Drug REpurposing with Artificial intelligence for Muscular disorderS

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            Abstract

            The World Health Organization highlights a critical need for treatments in rare diseases, with less than 6% currently having approved therapies. The EU-funded DREAMS project aims to discover therapies for five rare neuromuscular disorders with similar pathophysiological traits. To do so we will generate a collection of induced pluripotent stem cells from patients affected with Duchenne muscular dystrophy, Pompe disease, DNM2 centronuclear myopathy, Danon disease and Emery-Dreifuss muscular dystrophy. Following differentiation into skeletal muscle cells, these iPS cells will be used to discover common biomarkers across these disorders. A particular interest will be given to autophagic dysregulations that have previously been reported as a target of interest in those diseases. In DREAMS, we will also develop phenotypic and mechanism based screening approaches to test a library of 4000 drugs. Hits mechanism of action will be investigated using AI based target deconvolution algorithms and the best candidates will be evaluated in preclinical animal models of these diseases. Beyond the identification of repurposable drugs for these five diseases, DREAMS propose an innovative platform combining pluripotent stem cells, drug screening and AI usable to find treatments to other groups of diseases of genetic origin.

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            Author and article information

            Conference
            REPO4EU
            5 June 2024
            Affiliations
            [1 ] CECS/I-Stem, Evry, France;
            [2 ] Kantify, Brussels, Belgium;
            [3 ] Coimbra Universidad, Coimbra, Portugal ( https://ror.org/04z8k9a98)
            [4 ] Samsara Therapeutics, London, UK;
            [5 ] Technion, Haifa, Israel ( https://ror.org/03qryx823)
            [6 ] Inserm, Paris, France ( https://ror.org/02vjkv261)
            [7 ] Zabala innovation, Barcelona, Spain;
            [8 ] APHP, Paris, France;
            [9 ] AFM-Téléthon Evry, France;
            Article
            10.58647/REXPO.24007
            a291d076-1e5e-48b0-ae53-78eda6c4e2f8
            Copyright @ 2024

            Published under Creative Commons Attribution 4.0 International ( CC BY 4.0). Users are allowed to share (copy and redistribute the material in any medium or format) and adapt (remix, transform, and build upon the material for any purpose, even commercially), as long as the authors and the publisher are explicitly identified and properly acknowledged as the original source.

            RExPO24
            3
            Munich, Germany
            3-5 July 2024
            History
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            REPO4EU


            The datasets generated during and/or analysed during the current study are available from the corresponding author on reasonable request.
            Drug screening,Stem cells,Target ID,Myopathies

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