Hematopoietic stem cell gene therapy for metachromatic leukodystrophy and other lysosomal storage diseases

2009 ◽  
Author(s):  
A. Biffi
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Metachromatic Leukodystrophy ◽  
Lysosomal Storage Diseases ◽  
Lysosomal Storage ◽  
Hematopoietic Stem ◽  
Storage Diseases ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy

2021 ◽  
Author(s):  
Justin S. Antony ◽  
Alberto Daniel-Moreno ◽  
Andrés Lamsfus-Calle ◽  
Janani Raju ◽  
Merve Kaftancioglu ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Metachromatic Leukodystrophy ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

scholarly journals Hematopoietic Stem Cell Gene Therapy for the Multisystemic Lysosomal Storage Disorder Cystinosis

2013 ◽  
Vol 21 (2) ◽  
pp. 433-444 ◽  
Author(s):  
Frank Harrison ◽  
Brian A Yeagy ◽  
Celine J Rocca ◽  
Donald B Kohn ◽  
Daniel R Salomon ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Lysosomal Storage Disorder ◽  
Lysosomal Storage ◽  
Hematopoietic Stem ◽  
Storage Disorder ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy
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