Enzyme Replacement Therapy Ameliorates Multiple Symptoms of Murine Homocystinuria.

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第一作者： Tomas,Majtan

第一单位： Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO 80045, USA. Electronic address: tomas.majtan@ucdenver.edu.

作者： Tomas,Majtan ^[1] ; Wendell,Jones ^[2] ; Jakub,Krijt ^[3] ; Insun,Park ^[4] ; Warren D,Kruger ^[5] ; Viktor,Kožich ^[3] ; Steven,Bassnett ^[2] ; Erez M,Bublil ^[6] ; Jan P,Kraus ^[7]

作者单位： Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO 80045, USA. Electronic address: tomas.majtan@ucdenver.edu. ^[1] Department of Ophthalmology and Visual Sciences, Washington University School of Medicine, St. Louis, MO 63110, USA. ^[2] Department of Pediatrics and Adolescent Medicine, Charles University-First Faculty of Medicine and General University Hospital in Prague, Prague 12808, Czech Republic. ^[3] Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO 80045, USA. ^[4] Cancer Biology Program, Fox Chase Cancer Center, Philadelphia, PA 19111, USA. ^[5] Orphan Technologies, Ltd., Rapperswil 8640, Switzerland. ^[6] Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO 80045, USA. Electronic address: jan.kraus@ucdenver.edu. ^[7]

关键词 PEGylation alopecia bone density cystathionine beta-synthase enzyme replacement eye defect homocysteine inborn error of metabolism metabolomics preclinical studies