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A Promising Future for Stem-Cell-Based Therapies in Muscular Dystrophies—In Vitro and In Vivo Treatments to Boost Cellular Engraftment
Muscular dystrophies (MD) are a group of genetic diseases that lead to skeletal muscle wasting and may affect many organs (multisystem). Unfortunately, no curative therapies are available at present for MD patients, and current treatments mainly address the symptoms. Thus, stem-cell-based therapies...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6861917/ https://www.ncbi.nlm.nih.gov/pubmed/31683627 http://dx.doi.org/10.3390/ijms20215433 |
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author | Gois Beghini, Daniela Iwao Horita, Samuel Monteiro da Fonseca Cardoso, Liana Anastacio Alves, Luiz Nagaraju, Kanneboyina Henriques-Pons, Andrea |
author_facet | Gois Beghini, Daniela Iwao Horita, Samuel Monteiro da Fonseca Cardoso, Liana Anastacio Alves, Luiz Nagaraju, Kanneboyina Henriques-Pons, Andrea |
author_sort | Gois Beghini, Daniela |
collection | PubMed |
description | Muscular dystrophies (MD) are a group of genetic diseases that lead to skeletal muscle wasting and may affect many organs (multisystem). Unfortunately, no curative therapies are available at present for MD patients, and current treatments mainly address the symptoms. Thus, stem-cell-based therapies may present hope for improvement of life quality and expectancy. Different stem cell types lead to skeletal muscle regeneration and they have potential to be used for cellular therapies, although with several limitations. In this review, we propose a combination of genetic, biochemical, and cell culture treatments to correct pathogenic genetic alterations and to increase proliferation, dispersion, fusion, and differentiation into new or hybrid myotubes. These boosted stem cells can also be injected into pretreate recipient muscles to improve engraftment. We believe that this combination of treatments targeting the limitations of stem-cell-based therapies may result in safer and more efficient therapies for MD patients. Matricryptins have also discussed. |
format | Online Article Text |
id | pubmed-6861917 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-68619172019-12-05 A Promising Future for Stem-Cell-Based Therapies in Muscular Dystrophies—In Vitro and In Vivo Treatments to Boost Cellular Engraftment Gois Beghini, Daniela Iwao Horita, Samuel Monteiro da Fonseca Cardoso, Liana Anastacio Alves, Luiz Nagaraju, Kanneboyina Henriques-Pons, Andrea Int J Mol Sci Review Muscular dystrophies (MD) are a group of genetic diseases that lead to skeletal muscle wasting and may affect many organs (multisystem). Unfortunately, no curative therapies are available at present for MD patients, and current treatments mainly address the symptoms. Thus, stem-cell-based therapies may present hope for improvement of life quality and expectancy. Different stem cell types lead to skeletal muscle regeneration and they have potential to be used for cellular therapies, although with several limitations. In this review, we propose a combination of genetic, biochemical, and cell culture treatments to correct pathogenic genetic alterations and to increase proliferation, dispersion, fusion, and differentiation into new or hybrid myotubes. These boosted stem cells can also be injected into pretreate recipient muscles to improve engraftment. We believe that this combination of treatments targeting the limitations of stem-cell-based therapies may result in safer and more efficient therapies for MD patients. Matricryptins have also discussed. MDPI 2019-10-31 /pmc/articles/PMC6861917/ /pubmed/31683627 http://dx.doi.org/10.3390/ijms20215433 Text en © 2019 by the authors. 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 (http://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Gois Beghini, Daniela Iwao Horita, Samuel Monteiro da Fonseca Cardoso, Liana Anastacio Alves, Luiz Nagaraju, Kanneboyina Henriques-Pons, Andrea A Promising Future for Stem-Cell-Based Therapies in Muscular Dystrophies—In Vitro and In Vivo Treatments to Boost Cellular Engraftment |
title | A Promising Future for Stem-Cell-Based Therapies in Muscular Dystrophies—In Vitro and In Vivo Treatments to Boost Cellular Engraftment |
title_full | A Promising Future for Stem-Cell-Based Therapies in Muscular Dystrophies—In Vitro and In Vivo Treatments to Boost Cellular Engraftment |
title_fullStr | A Promising Future for Stem-Cell-Based Therapies in Muscular Dystrophies—In Vitro and In Vivo Treatments to Boost Cellular Engraftment |
title_full_unstemmed | A Promising Future for Stem-Cell-Based Therapies in Muscular Dystrophies—In Vitro and In Vivo Treatments to Boost Cellular Engraftment |
title_short | A Promising Future for Stem-Cell-Based Therapies in Muscular Dystrophies—In Vitro and In Vivo Treatments to Boost Cellular Engraftment |
title_sort | promising future for stem-cell-based therapies in muscular dystrophies—in vitro and in vivo treatments to boost cellular engraftment |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6861917/ https://www.ncbi.nlm.nih.gov/pubmed/31683627 http://dx.doi.org/10.3390/ijms20215433 |
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