scholarly journals Induction of Tolerance to Human Arylsulfatase A in a Mouse Model of Metachromatic Leukodystrophy

2007 ◽  
Vol 13 (9-10) ◽  
pp. 471-479 ◽  
Author(s):  
Ulrich Matzner ◽  
Frank Matthes ◽  
Eva Herbst ◽  
Renate Lüllmann-Rauch ◽  
Zsuzsanna Callaerts-Vegh ◽  
...  
Keyword(s):  
Mouse Model ◽  
Metachromatic Leukodystrophy ◽  
Arylsulfatase A ◽  
Human Arylsulfatase ◽  
Induction Of Tolerance

scholarly journals AAV1 Mediated Co-expression of Formylglycine-Generating Enzyme and Arylsulfatase A Efficiently Corrects Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy

2007 ◽  
Vol 15 (1) ◽  
pp. 38-43 ◽  
Author(s):  
Toshiyuki Kurai ◽  
Sanae Hisayasu ◽  
Ryo Kitagawa ◽  
Makoto Migita ◽  
Hidenori Suzuki ◽  
...  
Keyword(s):  
Mouse Model ◽  
Metachromatic Leukodystrophy ◽  
Arylsulfatase A

scholarly journals Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial

2020 ◽  
Vol 131 (1-2) ◽  
pp. 235-244 ◽  
Author(s):  
Christine í Dali ◽  
Caroline Sevin ◽  
Ingeborg Krägeloh-Mann ◽  
Roberto Giugliani ◽  
Norio Sakai ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Metachromatic Leukodystrophy ◽  
Phase 1 ◽  
Arylsulfatase A ◽  
Human Arylsulfatase

Intrathecal delivery of recombinant human arylsulfatase A in children with late-infantile metachromatic leukodystrophy: an update following extended treatment

2017 ◽  
Vol 120 (1-2) ◽  
pp. S121 ◽  
Author(s):  
Caroline Sevin ◽  
Christine Dali ◽  
Roberto Giugliani ◽  
Joachim Riethmüller ◽  
Christopher Troedson ◽  
...  
Keyword(s):  
Metachromatic Leukodystrophy ◽  
Arylsulfatase A ◽  
Extended Treatment ◽  
Human Arylsulfatase

Intrathecal delivery of recombinant human arylsulfatase A in children with late-infantile metachromatic leukodystrophy

2016 ◽  
Vol 117 (2) ◽  
pp. S38 ◽  
Author(s):  
Christine Dali ◽  
Caroline Sevin ◽  
Joachim Riethmüller ◽  
Roberto Giugliani ◽  
Christopher Troedson ◽  
...  
Keyword(s):  
Metachromatic Leukodystrophy ◽  
Arylsulfatase A ◽  
Human Arylsulfatase

scholarly journals Arylsulfatase A Overexpressing Human iPSC-derived Neural Cells Reduce CNS Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy

2015 ◽  
Vol 23 (9) ◽  
pp. 1519-1531 ◽  
Author(s):  
Jonas Doerr ◽  
Annika Böckenhoff ◽  
Benjamin Ewald ◽  
Julia Ladewig ◽  
Matthias Eckhardt ◽  
...  
Keyword(s):  
Mouse Model ◽  
Metachromatic Leukodystrophy ◽  
Neural Cells ◽  
Arylsulfatase A ◽  
Human Ipsc
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