Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?

Mucopolysaccharidoses (MPSs) are a group of lysosomal storage disorders (LSDs). The increasing interest in newborn screening procedures for LSDs underlines the need for alternative cellular and gene therapy approaches to be developed during the perinatal period, supporting the treatment of MPS patie...

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Detalles Bibliográficos
Autores principales: Tomatsu, Shunji, Azario, Isabella, Sawamoto, Kazuki, Pievani, Alice Silvia, Biondi, Andrea, Serafini, Marta
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Netherlands 2015
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4754332/
https://www.ncbi.nlm.nih.gov/pubmed/26578156
http://dx.doi.org/10.1007/s10545-015-9900-2

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