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Development of therapies for rare genetic disorders of GPX4: roadmap and opportunities.

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Title: Development of therapies for rare genetic disorders of GPX4: roadmap and opportunities.
Authors: Cheff DM; National Center for Advancing Translational Sciences, National Institutes of Health, Rockville, MD, USA.; Division of Biochemistry, Department of Medical Biochemistry and Biophysics, Karolinska Institutet, 171 77, Stockholm, Sweden., Muotri AR; Department of Pediatrics, University of California, San Diego, San Diego, CA, USA.; Department of Cellular and Molecular Medicine, University of California, San Diego, San Diego, CA, USA., Stockwell BR; Department of Biological Sciences, Columbia University, New York, NY, USA.; Department of Chemistry, Columbia University, New York, NY, USA., Schmidt EE; Department of Microbiology and Immunology, Montana State University, Bozeman, MT, USA., Ran Q; Department of Cell Systems and Anatomy, University of Texas Health Science Center, San Antonio, San Antonio, TX, USA.; Research and Development Service, South Texas Veterans Health Care System, San Antonio, TX, USA., Kartha RV; Department of Experimental and Clinical Pharmacology, Center for Orphan Drug Research, College of Pharmacy, University of Minnesota, Minneapolis, MN, USA., Johnson SC; Department of Neurology, University of Washington, Seattle, WA, USA.; Department of Anesthesiology and Pain Medicine, University of Washington, Seattle, WA, USA.; Center for Integrative Brain Research, Seattle Children's Research Institute, Seattle, WA, USA., Mittal P; In-Depth Genomics, Bellevue, WA, USA., Arnér ESJ; Division of Biochemistry, Department of Medical Biochemistry and Biophysics, Karolinska Institutet, 171 77, Stockholm, Sweden.; Department of Selenoprotein Research, National Institute of Oncology, Budapest, 1521, Hungary., Wigby KM; Department of Pediatrics, Division of Genetics, San Diego and Rady Children's Hospital-San Diego, University of California, San Diego, CA, USA.; Rady Children's Institute for Genomic Medicine, San Diego, CA, USA., Hall MD; National Center for Advancing Translational Sciences, National Institutes of Health, Rockville, MD, USA., Ramesh SK; CureGPX4.org, Seattle, WA, USA. sanath@GPX4.org.
Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2021 Oct 23; Vol. 16 (1), pp. 446. Date of Electronic Publication: 2021 Oct 23.
Publication Type: Journal Article; Research Support, N.I.H., Intramural; Research Support, Non-U.S. Gov't; Review
Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Development of therapies for rare genetic disorders of GPX4: roadmap and opportunities.
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  Data: <searchLink fieldCode="AU" term="%22Cheff+DM%22">Cheff DM</searchLink>; National Center for Advancing Translational Sciences, National Institutes of Health, Rockville, MD, USA.; Division of Biochemistry, Department of Medical Biochemistry and Biophysics, Karolinska Institutet, 171 77, Stockholm, Sweden.<br /><searchLink fieldCode="AU" term="%22Muotri+AR%22">Muotri AR</searchLink>; Department of Pediatrics, University of California, San Diego, San Diego, CA, USA.; Department of Cellular and Molecular Medicine, University of California, San Diego, San Diego, CA, USA.<br /><searchLink fieldCode="AU" term="%22Stockwell+BR%22">Stockwell BR</searchLink>; Department of Biological Sciences, Columbia University, New York, NY, USA.; Department of Chemistry, Columbia University, New York, NY, USA.<br /><searchLink fieldCode="AU" term="%22Schmidt+EE%22">Schmidt EE</searchLink>; Department of Microbiology and Immunology, Montana State University, Bozeman, MT, USA.<br /><searchLink fieldCode="AU" term="%22Ran+Q%22">Ran Q</searchLink>; Department of Cell Systems and Anatomy, University of Texas Health Science Center, San Antonio, San Antonio, TX, USA.; Research and Development Service, South Texas Veterans Health Care System, San Antonio, TX, USA.<br /><searchLink fieldCode="AU" term="%22Kartha+RV%22">Kartha RV</searchLink>; Department of Experimental and Clinical Pharmacology, Center for Orphan Drug Research, College of Pharmacy, University of Minnesota, Minneapolis, MN, USA.<br /><searchLink fieldCode="AU" term="%22Johnson+SC%22">Johnson SC</searchLink>; Department of Neurology, University of Washington, Seattle, WA, USA.; Department of Anesthesiology and Pain Medicine, University of Washington, Seattle, WA, USA.; Center for Integrative Brain Research, Seattle Children's Research Institute, Seattle, WA, USA.<br /><searchLink fieldCode="AU" term="%22Mittal+P%22">Mittal P</searchLink>; In-Depth Genomics, Bellevue, WA, USA.<br /><searchLink fieldCode="AU" term="%22Arnér+ESJ%22">Arnér ESJ</searchLink>; Division of Biochemistry, Department of Medical Biochemistry and Biophysics, Karolinska Institutet, 171 77, Stockholm, Sweden.; Department of Selenoprotein Research, National Institute of Oncology, Budapest, 1521, Hungary.<br /><searchLink fieldCode="AU" term="%22Wigby+KM%22">Wigby KM</searchLink>; Department of Pediatrics, Division of Genetics, San Diego and Rady Children's Hospital-San Diego, University of California, San Diego, CA, USA.; Rady Children's Institute for Genomic Medicine, San Diego, CA, USA.<br /><searchLink fieldCode="AU" term="%22Hall+MD%22">Hall MD</searchLink>; National Center for Advancing Translational Sciences, National Institutes of Health, Rockville, MD, USA.<br /><searchLink fieldCode="AU" term="%22Ramesh+SK%22">Ramesh SK</searchLink>; CureGPX4.org, Seattle, WA, USA. sanath@GPX4.org.
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  Data: <searchLink fieldCode="JN" term="%22101266602%22">Orphanet journal of rare diseases</searchLink> [Orphanet J Rare Dis] 2021 Oct 23; Vol. 16 (1), pp. 446. <i>Date of Electronic Publication: </i>2021 Oct 23.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22BioMed+Central%22">BioMed Central </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>101266602 <i>Publication Model: </i>Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1750-1172 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2217501172%22">17501172 </searchLink><i>NLM ISO Abbreviation: </i>Orphanet J Rare Dis <i>Subsets: </i>MEDLINE
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