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