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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Detalles Bibliográficos
Autores principales: Fraldi, Alessandro, Serafini, Marta, Sorrentino, Nicolina Cristina, Gentner, Bernhard, Aiuti, Alessandro, Bernardo, Maria Ester
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6238250/
https://www.ncbi.nlm.nih.gov/pubmed/30442177
http://dx.doi.org/10.1186/s13052-018-0565-y

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6238250/
https://www.ncbi.nlm.nih.gov/pubmed/30442177
http://dx.doi.org/10.1186/s13052-018-0565-y

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