Date: 2015-08-03
Type of information: Publication of results in a medical journal
phase:
Announcement: publication of results in Nature Biotechnology
Company: Anagenesis Biotechnologies (France)
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Action mechanism:
Disease:
Therapeutic area: Neuromuscular diseases - Regenerative medicine
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Trial details:
Latest
news: * On August 3, 2015, Anagenesis Biotechnologies announced a key publication in Nature Biotechnology. The article is entitled “Differentiating embryonic stem cells to muscle fibers to model Duchenne Muscular Dystrophy” by Jérôme Chal et al. from the lab of Pr Olivier Pourquié (the two scientists inventors of Anagenesis Biotechnologies technology) together with co-authors from Anagenesis Biotechnologies.
Key cell types including skeletal muscle have proven difficult to differentiate in vitro from pluripotent cells. By recapitulating the development of skeletal muscle in chemically defined conditions, the authors have shown that myogenic cells can be produced in vitro from mouse and human embryonic (ES) and reprogrammed (iPS) stem cells without genetic manipulations or cell sorting. Anagenesis' technology allows for efficient production of striated contractile muscle fibers and of Pax7-positive cells with satellite cell characteristics. The satellite-like cells produced in vitro were able to generate dystrophin-positive fibers when grafted into muscles from dystrophin-deficient mdx mice in vivo, thus providing a first proof of concept for cell therapy protocols for Duchenne Muscular Dystrophy.
Differentiated ES cells derived from mdx mice also exhibit a striking branched phenotype resembling that described in vivo, thus providing an attractive model to study the origin of the pathological defects associated with Duchenne muscular dystrophy. This work also opens the possibility to develop in vitro models to study the pathology of muscular dystrophies as well as high throughput assays based on myogenic cells for drug screening.
