Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches

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 transplantation...

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書目詳細資料
發表在:Ital J Pediatr
Main Authors: Fraldi, Alessandro, Serafini, Marta, Sorrentino, Nicolina Cristina, Gentner, Bernhard, Aiuti, Alessandro, Bernardo, Maria Ester
格式: Artigo
語言:Inglês
出版: BioMed Central 2018
主題:
Review
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6238250/
https://ncbi.nlm.nih.gov/pubmed/30442177
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13052-018-0565-y
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