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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Bibliographic Details
Main Authors: Tomatsu, Shunji, Azario, Isabella, Sawamoto, Kazuki, Pievani, Alice Silvia, Biondi, Andrea, Serafini, Marta
Format: Online
Language:English
Published: Springer Netherlands 2015
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4754332/

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