Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of α-glucosidase in Pompe disease-Reference-Cited by-同舟云学术

Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of α-glucosidase in Pompe disease

Published:2009-04 Issue:4 Volume:11 Page:279-287
ISSN:1099-498X
Container-title:The Journal of Gene Medicine
language:en
Short-container-title:J. Gene Med.

Author:

Douillard-Guilloux Gaëlle,Richard Emmanuel,Batista Lionel,Caillaud Catherine

Publisher

Wiley

Subject

Genetics(clinical),Drug Discovery,Genetics,Molecular Biology,Molecular Medicine

Reference59 articles.

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4. Infantile-Onset Pompe Disease Natural History Study Group. A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease;Kishnani;J Pediatr,2006

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