Mucopolysaccharidoses (MPSs) are a group of lysosomal\nstorage disorders (LSDs). The increasing interest in\nnewborn screening procedures for LSDs underlines the need\nfor alternative cellular and gene therapy approaches to be developed\nduring the perinatal period, supporting the treatment\nof MPS patients before the onset of clinical signs and symptoms.\nThe rationale for considering these early therapies results\nfromthe clinical experience in the treatment ofMPSs and\nother genetic disorders. The normal or gene-corrected hematopoiesis\ntransplanted in patients can produce the missing protein\nat levels sufficient to improve and/or halt the diseaserelated\nabnormalities. However, these current therapies are\nonly partially successful, probably due to the limited efficacy\nof the protein provided through the hematopoiesis. An alternative\nexplanation is that the time at which the cellular or gene\ntherapy procedures are performed could be too late to prevent\npre-existing or progressive organ damage. Considering these\naspects, in the last several years, novel cellular and gene therapy\napproaches have been tested in different animal models at\nbirth, a highly early stage, showing that precocious treatment\nis critical to prevent long-term pathological consequences.\nThis review provides insights into the state-of-art accomplishments\nmade with neonatal cellular and gene-based therapies\nand the major barriers that need to be overcome before they\ncan be implemented in the medical community.
Loading....