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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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Shranjeno v:
Bibliografske podrobnosti
izdano v:J Inherit Metab Dis
Main Authors: Tomatsu, Shunji, Azario, Isabella, Sawamoto, Kazuki, Pievani, Alice Silvia, Biondi, Andrea, Serafini, Marta
Format: Artigo
Jezik:Inglês
Izdano: Springer Netherlands 2015
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC4754332/
https://ncbi.nlm.nih.gov/pubmed/26578156
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-015-9900-2
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