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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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/

id	pubmed-4754332
recordtype	oai_dc
spelling	pubmed-47543322016-02-25 Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better? Tomatsu, Shunji Azario, Isabella Sawamoto, Kazuki Pievani, Alice Silvia Biondi, Andrea Serafini, Marta Review 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 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-corrected hematopoiesis transplanted in patients can produce the missing protein at levels sufficient to improve and/or halt the disease-related 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-existing 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-term pathological consequences. This review provides insights into the state-of-art accomplishments made with neonatal cellular and gene-based therapies and the major barriers that need to be overcome before they can be implemented in the medical community. Springer Netherlands 2015-11-17 2016 /pmc/articles/PMC4754332/ /pubmed/26578156 http://dx.doi.org/10.1007/s10545-015-9900-2 Text en © The Author(s) 2015 Open Access This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made.
repository_type	Open Access Journal
institution_category	Foreign Institution
institution	US National Center for Biotechnology Information
building	NCBI PubMed
collection	Online Access
language	English
format	Online
author	Tomatsu, Shunji Azario, Isabella Sawamoto, Kazuki Pievani, Alice Silvia Biondi, Andrea Serafini, Marta
spellingShingle	Tomatsu, Shunji Azario, Isabella Sawamoto, Kazuki Pievani, Alice Silvia Biondi, Andrea Serafini, Marta Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?
author_facet	Tomatsu, Shunji Azario, Isabella Sawamoto, Kazuki Pievani, Alice Silvia Biondi, Andrea Serafini, Marta
author_sort	Tomatsu, Shunji
title	Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?
title_short	Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?
title_full	Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?
title_fullStr	Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?
title_full_unstemmed	Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?
title_sort	neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?
description	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 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-corrected hematopoiesis transplanted in patients can produce the missing protein at levels sufficient to improve and/or halt the disease-related 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-existing 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-term pathological consequences. This review provides insights into the state-of-art accomplishments made with neonatal cellular and gene-based therapies and the major barriers that need to be overcome before they can be implemented in the medical community.
publisher	Springer Netherlands
publishDate	2015
url	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4754332/
_version_	1613538812251602944

Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?

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