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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...

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Autores principales: Gois Beghini, Daniela, Iwao Horita, Samuel, Monteiro da Fonseca Cardoso, Liana, Anastacio Alves, Luiz, Nagaraju, Kanneboyina, Henriques-Pons, Andrea
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2019
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.
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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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