Gene and Cell Therapy for Muscular Dystrophies: Are We Getting There?

In the last few years, significant advances have occurred in the preclinical and clinical work toward gene and cell therapy for muscular dystrophy. At the time of this writing, several trials are ongoing and more are expected to start. It is thus a time of expectation, even though many hurdles remai...

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Autores principales: Galli, Francesco, Bragg, Laricia, Meggiolaro, Linda, Rossi, Maira, Caffarini, Miriam, Naz, Naila, Santoleri, Sabrina, Cossu, Giulio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2018
Materias:
Reviews
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6211823/
https://www.ncbi.nlm.nih.gov/pubmed/30132372
http://dx.doi.org/10.1089/hum.2018.151
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author Galli, Francesco
Bragg, Laricia
Meggiolaro, Linda
Rossi, Maira
Caffarini, Miriam
Naz, Naila
Santoleri, Sabrina
Cossu, Giulio
author_facet Galli, Francesco
Bragg, Laricia
Meggiolaro, Linda
Rossi, Maira
Caffarini, Miriam
Naz, Naila
Santoleri, Sabrina
Cossu, Giulio
author_sort Galli, Francesco
collection PubMed
description In the last few years, significant advances have occurred in the preclinical and clinical work toward gene and cell therapy for muscular dystrophy. At the time of this writing, several trials are ongoing and more are expected to start. It is thus a time of expectation, even though many hurdles remain and it is unclear whether they will be overcome with current strategies or if further improvements will be necessary.
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spelling pubmed-62118232018-11-02 Gene and Cell Therapy for Muscular Dystrophies: Are We Getting There? Galli, Francesco Bragg, Laricia Meggiolaro, Linda Rossi, Maira Caffarini, Miriam Naz, Naila Santoleri, Sabrina Cossu, Giulio Hum Gene Ther Reviews In the last few years, significant advances have occurred in the preclinical and clinical work toward gene and cell therapy for muscular dystrophy. At the time of this writing, several trials are ongoing and more are expected to start. It is thus a time of expectation, even though many hurdles remain and it is unclear whether they will be overcome with current strategies or if further improvements will be necessary. Mary Ann Liebert, Inc., publishers 2018-10-01 2018-10-12 /pmc/articles/PMC6211823/ /pubmed/30132372 http://dx.doi.org/10.1089/hum.2018.151 Text en © Francesco Galli et al. 2018; Published by Mary Ann Liebert, Inc. This Open Access article is distributed under the terms of the Creative Commons License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Reviews
Galli, Francesco
Bragg, Laricia
Meggiolaro, Linda
Rossi, Maira
Caffarini, Miriam
Naz, Naila
Santoleri, Sabrina
Cossu, Giulio
Gene and Cell Therapy for Muscular Dystrophies: Are We Getting There?
title Gene and Cell Therapy for Muscular Dystrophies: Are We Getting There?
title_full Gene and Cell Therapy for Muscular Dystrophies: Are We Getting There?
title_fullStr Gene and Cell Therapy for Muscular Dystrophies: Are We Getting There?
title_full_unstemmed Gene and Cell Therapy for Muscular Dystrophies: Are We Getting There?
title_short Gene and Cell Therapy for Muscular Dystrophies: Are We Getting There?
title_sort gene and cell therapy for muscular dystrophies: are we getting there?
topic Reviews
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6211823/
https://www.ncbi.nlm.nih.gov/pubmed/30132372
http://dx.doi.org/10.1089/hum.2018.151
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