New work has provided insight into the molecular pathways that underlie the liver disease that is associated with α1-antitrypsin deficiency, and has identified a novel approach to cell-based therapy.
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D.A.L. is funded by the Engineering and Physical Science Research Council, GlaxoSmithKline, the Medical Research Council, the Rosetrees Trust, the University College London Hospital National Institute for Health Research Biomedical Research Centre, and the Wellcome Trust. He is working with GlaxoSmithKline to develop small molecules that block the intracellular polymerization of α1-antitrypsin. S.T.R. is funded by the Medical Research Council and holds shares as a co-scientific founder in the company DefiniGen.
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Rashid, S., Lomas, D. Taking out the JuNK to treat α1-antitrypsin deficiency. Nat Rev Gastroenterol Hepatol 14, 201–202 (2017). https://doi.org/10.1038/nrgastro.2017.22
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DOI: https://doi.org/10.1038/nrgastro.2017.22