Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art
The need for long-lasting and transformative therapies for mucopolysaccharidoses (MPS) cannot be understated. Currently, many forms of MPS lack a specific treatment and in other cases available therapies, such as enzyme replacement therapy (ERT), do not reach important areas such as the central nerv...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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MDPI
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8430935/ https://www.ncbi.nlm.nih.gov/pubmed/34502108 http://dx.doi.org/10.3390/ijms22179200 |
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author | de Castro, María José del Toro, Mireia Giugliani, Roberto Couce, María Luz |
author_facet | de Castro, María José del Toro, Mireia Giugliani, Roberto Couce, María Luz |
author_sort | de Castro, María José |
collection | PubMed |
description | The need for long-lasting and transformative therapies for mucopolysaccharidoses (MPS) cannot be understated. Currently, many forms of MPS lack a specific treatment and in other cases available therapies, such as enzyme replacement therapy (ERT), do not reach important areas such as the central nervous system (CNS). The advent of newborn screening procedures represents a major step forward in early identification and treatment of individuals with MPS. However, the treatment of brain disease in neuronopathic MPS has been a major challenge to date, mainly because the blood brain barrier (BBB) prevents penetration of the brain by large molecules, including enzymes. Over the last years several novel experimental therapies for neuronopathic MPS have been investigated. Gene therapy and gene editing constitute potentially curative treatments. However, despite recent progress in the field, several considerations should be taken into account. This review focuses on the state of the art of in vivo and ex vivo gene therapy-based approaches targeting the CNS in neuronopathic MPS, discusses clinical trials conducted to date, and provides a vision for the future implications of these therapies for the medical community. Recent advances in the field, as well as limitations relating to efficacy, potential toxicity, and immunogenicity, are also discussed. |
format | Online Article Text |
id | pubmed-8430935 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-84309352021-09-11 Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art de Castro, María José del Toro, Mireia Giugliani, Roberto Couce, María Luz Int J Mol Sci Review The need for long-lasting and transformative therapies for mucopolysaccharidoses (MPS) cannot be understated. Currently, many forms of MPS lack a specific treatment and in other cases available therapies, such as enzyme replacement therapy (ERT), do not reach important areas such as the central nervous system (CNS). The advent of newborn screening procedures represents a major step forward in early identification and treatment of individuals with MPS. However, the treatment of brain disease in neuronopathic MPS has been a major challenge to date, mainly because the blood brain barrier (BBB) prevents penetration of the brain by large molecules, including enzymes. Over the last years several novel experimental therapies for neuronopathic MPS have been investigated. Gene therapy and gene editing constitute potentially curative treatments. However, despite recent progress in the field, several considerations should be taken into account. This review focuses on the state of the art of in vivo and ex vivo gene therapy-based approaches targeting the CNS in neuronopathic MPS, discusses clinical trials conducted to date, and provides a vision for the future implications of these therapies for the medical community. Recent advances in the field, as well as limitations relating to efficacy, potential toxicity, and immunogenicity, are also discussed. MDPI 2021-08-25 /pmc/articles/PMC8430935/ /pubmed/34502108 http://dx.doi.org/10.3390/ijms22179200 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review de Castro, María José del Toro, Mireia Giugliani, Roberto Couce, María Luz Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art |
title | Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art |
title_full | Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art |
title_fullStr | Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art |
title_full_unstemmed | Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art |
title_short | Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art |
title_sort | gene therapy for neuronopathic mucopolysaccharidoses: state of the art |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8430935/ https://www.ncbi.nlm.nih.gov/pubmed/34502108 http://dx.doi.org/10.3390/ijms22179200 |
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