Enzyme replacement therapy for Farber disease: Proof-of-concept studies in cells and mice.

He, Xingxuan; Dworski, Shaalee; Zhu, Changzhi; DeAngelis, Victor; Solyom, Alex; Medin, Jeffrey A; Simonaro, Calogera M; Schuchman, Edward H.

Afiliação

He X; Department of Genetics & Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, USA.
Dworski S; Institute of Medical Science, University of Toronto, Toronto, Canada.
Zhu C; Department of Genetics & Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, USA.
DeAngelis V; Department of Genetics & Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, USA.
Solyom A; Enzyvant Sciences, NY, New York, USA.
Medin JA; Institute of Medical Science, University of Toronto, Toronto, Canada; Departments of Pediatrics and Biochemistry, Medical College of Wisconsin, Milwaukee, WI, USA.
Simonaro CM; Department of Genetics & Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, USA.
Schuchman EH; Department of Genetics & Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, USA.

BBA Clin ; 7: 85-96, 2017 Jun.

Article em En | MEDLINE | ID: mdl-28275553