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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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| Publicado no: | Ital J Pediatr |
|---|---|
| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
BioMed Central
2018
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| Assuntos: | |
| Acesso em linha: | 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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