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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 patients before the onset of clinical signs and symptoms. The rationale for considering these early therapies results from the clinical experience in the treatment of MPSs and other genetic disorders. The normal or gene\u2010corrected hematopoiesis transplanted in patients can produce the missing protein at levels sufficient to improve and\/or halt the disease\u2010related abnormalities. However, these current therapies are only partially successful, probably due to the limited efficacy of the protein provided through the hematopoiesis. An alternative explanation is that the time at which the cellular or gene therapy procedures are performed could be too late to prevent pre\u2010existing or progressive organ damage. Considering these aspects, in the last several years, novel cellular and gene therapy approaches have been tested in different animal models at birth, a highly early stage, showing that precocious treatment is critical to prevent long\u2010term pathological consequences. This review provides insights into the state\u2010of\u2010art accomplishments made with neonatal cellular and gene\u2010based therapies and the major barriers that need to be overcome before they can be implemented in the medical community.<\/jats:p>","DOI":"10.1007\/s10545-015-9900-2","type":"journal-article","created":{"date-parts":[[2015,11,17]],"date-time":"2015-11-17T13:18:50Z","timestamp":1447766330000},"page":"189-202","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":23,"title":["Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?"],"prefix":"10.1002","volume":"39","author":[{"given":"Shunji","family":"Tomatsu","sequence":"first","affiliation":[{"name":"Department of Biomedical Research Alfred I. duPont Institute Hospital for Children Wilmington DE USA"},{"name":"Skeletal Dysplasia Lab, Department of Biomedical Research Nemours\/Alfred I. duPont Hospital for Children 1600 Rockland Rd. 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