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...
| Main Authors: | , , , , , |
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| Format: | Online |
| Language: | English |
| Published: |
Springer Netherlands
2015
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| Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4754332/ |