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GLA-modified RNA treatment lowers GB3 levels in iPSC-derived cardiomyocytes from Fabry-affected individuals.
Ter Huurne, Menno; Parker, Benjamin L; Liu, Ning Qing; Qian, Elizabeth Ling; Vivien, Celine; Karavendzas, Kathy; Mills, Richard J; Saville, Jennifer T; Abu-Bonsrah, Dad; Wise, Andrea F; Hudson, James E; Talbot, Andrew S; Finn, Patrick F; Martini, Paolo G V; Fuller, Maria; Ricardo, Sharon D; Watt, Kevin I; Nicholls, Kathy M; Porrello, Enzo R; Elliott, David A.
Affiliation
  • Ter Huurne M; Murdoch Children's Research Institute, The Royal Children's Hospital, Melbourne, VIC, Australia; The Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Murdoch Children's Research Institute, Melbourne, VIC, Australia; Department of Paediatrics, University of Melbourne, Melbourne, VIC, Au
  • Parker BL; Department of Anatomy and Physiology, University of Melbourne, Melbourne, VIC, Australia; Centre for Muscle Research, University of Melbourne, Melbourne, VIC, Australia.
  • Liu NQ; Department of Hematology, Erasmus Medical Center (MC) Cancer Institute, Rotterdam, the Netherlands.
  • Qian EL; Murdoch Children's Research Institute, The Royal Children's Hospital, Melbourne, VIC, Australia.
  • Vivien C; Murdoch Children's Research Institute, The Royal Children's Hospital, Melbourne, VIC, Australia.
  • Karavendzas K; Murdoch Children's Research Institute, The Royal Children's Hospital, Melbourne, VIC, Australia.
  • Mills RJ; Murdoch Children's Research Institute, The Royal Children's Hospital, Melbourne, VIC, Australia; The Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Murdoch Children's Research Institute, Melbourne, VIC, Australia; QIMR Berghofer Medical Research Institute, Brisbane, QLD 4006, Austral
  • Saville JT; Genetics and Molecular Pathology, SA Pathology at Women's and Children's Hospital and Adelaide Medical School, University of Adelaide, Adelaide, SA, Australia.
  • Abu-Bonsrah D; Murdoch Children's Research Institute, The Royal Children's Hospital, Melbourne, VIC, Australia.
  • Wise AF; Department of Pharmacology, Biomedicine Discovery Institute, Monash University, Clayton, VIC, Australia.
  • Hudson JE; QIMR Berghofer Medical Research Institute, Brisbane, QLD 4006, Australia.
  • Talbot AS; Department of Nephrology, The Royal Melbourne Hospital and Department of Medicine (RMH), University of Melbourne, Parkville, VIC, Australia.
  • Finn PF; Rare Diseases Research, Moderna Inc., 200 Technology Sq., Cambridge, MA, USA.
  • Martini PGV; Rare Diseases Research, Moderna Inc., 200 Technology Sq., Cambridge, MA, USA.
  • Fuller M; Genetics and Molecular Pathology, SA Pathology at Women's and Children's Hospital and Adelaide Medical School, University of Adelaide, Adelaide, SA, Australia.
  • Ricardo SD; Department of Pharmacology, Biomedicine Discovery Institute, Monash University, Clayton, VIC, Australia.
  • Watt KI; Murdoch Children's Research Institute, The Royal Children's Hospital, Melbourne, VIC, Australia; The Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Murdoch Children's Research Institute, Melbourne, VIC, Australia; Department of Anatomy and Physiology, University of Melbourne, Melbour
  • Nicholls KM; Department of Pharmacology, Biomedicine Discovery Institute, Monash University, Clayton, VIC, Australia.
  • Porrello ER; Murdoch Children's Research Institute, The Royal Children's Hospital, Melbourne, VIC, Australia; The Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Murdoch Children's Research Institute, Melbourne, VIC, Australia; Department of Anatomy and Physiology, University of Melbourne, Melbour
  • Elliott DA; Murdoch Children's Research Institute, The Royal Children's Hospital, Melbourne, VIC, Australia; The Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Murdoch Children's Research Institute, Melbourne, VIC, Australia; Melbourne Centre for Cardiovascular Genomics and Regenerative Medicine
Am J Hum Genet ; 110(9): 1600-1605, 2023 09 07.
Article de En | MEDLINE | ID: mdl-37607539
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Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Maladie de Fabry / Cellules souches pluripotentes induites Limites: Humans Langue: En Journal: Am J Hum Genet Année: 2023 Type de document: Article Pays d'affiliation: Australie
Texte intégral
Ajouter à My VHL
Imprimer
XML
PubMed Links
Recherche sur Google

Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Maladie de Fabry / Cellules souches pluripotentes induites Limites: Humans Langue: En Journal: Am J Hum Genet Année: 2023 Type de document: Article Pays d'affiliation: Australie