Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches

Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches

Abstract Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders caused by a deficiency in lysosomal enzymes catalyzing the stepwise degradation of glycosaminoglycans (GAGs). The current therapeutic strategies of enzyme replacement therapy and allogeneic hematopoietic stem cell transp...

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Bibliographic Details
Main Authors: Alessandro Fraldi, Marta Serafini, Nicolina Cristina Sorrentino, Bernhard Gentner, Alessandro Aiuti, Maria Ester Bernardo
Format: Article
Language:English
Published: BMC 2018-11-01
Series:Italian Journal of Pediatrics
Online Access:http://link.springer.com/article/10.1186/s13052-018-0565-y

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http://link.springer.com/article/10.1186/s13052-018-0565-y

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