Combined therapies for Duchenne muscular dystrophy to optimize treatment efficacy

Gonzalo Cordova, Elisa Negroni, Claudio Cabello-Verrugio, Vincent Mouly, Capucine Trollet

Resultado de la investigación: Short survey

7 Citas (Scopus)

Resumen

Duchene Muscular Dystrophy (DMD) is the most frequent muscular dystrophy and one of the most severe due to the absence of the dystrophin protein. Typical pathological features include muscle weakness, muscle wasting, degeneration, and inflammation. At advanced stages DMD muscles present exacerbated extracellular matrix and fat accumulation. Recent progress in therapeutic approaches has allowed new strategies to be investigated, including pharmacological, gene-based and cell-based therapies. Gene and cell-based therapies are still limited by poor targeting and low efficiency in fibrotic dystrophic muscle, therefore it is increasingly evident that future treatments will have to include "combined therapies" to reach maximal efficiency. The scope of this mini-review is to provide an overview of the current literature on such combined therapies for DMD. By "combined therapies" we mean those that include both a therapy to correct the genetic defect and an additional one to address one of the secondary pathological features of the disease. In this mini-review, we will not provide a comprehensive view of the literature on therapies for DMD, since many such reviews already exist, but we will focus on the characteristics, efficiency, and potential of such combined therapeutic strategies that have been described so far for DMD.

Idioma originalEnglish
Número de artículo114
PublicaciónFrontiers in Genetics
Volumen9
N.ºAPR
DOI
EstadoPublished - 10 abr 2018

Huella dactilar

Duchenne Muscular Dystrophy
Muscular Dystrophies
Therapeutics
Cell- and Tissue-Based Therapy
Muscles
Dystrophin
Muscle Weakness
Genes
Extracellular Matrix
Fats
Pharmacology
Inflammation

ASJC Scopus subject areas

  • Molecular Medicine
  • Genetics
  • Genetics(clinical)

Citar esto

Cordova, Gonzalo ; Negroni, Elisa ; Cabello-Verrugio, Claudio ; Mouly, Vincent ; Trollet, Capucine. / Combined therapies for Duchenne muscular dystrophy to optimize treatment efficacy. En: Frontiers in Genetics. 2018 ; Vol. 9, N.º APR.
@article{e4a3a87142714759bd4b5042ec75e4b8,
title = "Combined therapies for Duchenne muscular dystrophy to optimize treatment efficacy",
abstract = "Duchene Muscular Dystrophy (DMD) is the most frequent muscular dystrophy and one of the most severe due to the absence of the dystrophin protein. Typical pathological features include muscle weakness, muscle wasting, degeneration, and inflammation. At advanced stages DMD muscles present exacerbated extracellular matrix and fat accumulation. Recent progress in therapeutic approaches has allowed new strategies to be investigated, including pharmacological, gene-based and cell-based therapies. Gene and cell-based therapies are still limited by poor targeting and low efficiency in fibrotic dystrophic muscle, therefore it is increasingly evident that future treatments will have to include {"}combined therapies{"} to reach maximal efficiency. The scope of this mini-review is to provide an overview of the current literature on such combined therapies for DMD. By {"}combined therapies{"} we mean those that include both a therapy to correct the genetic defect and an additional one to address one of the secondary pathological features of the disease. In this mini-review, we will not provide a comprehensive view of the literature on therapies for DMD, since many such reviews already exist, but we will focus on the characteristics, efficiency, and potential of such combined therapeutic strategies that have been described so far for DMD.",
keywords = "Atrophy, Cell therapy, Duchenne muscular dystrophy, Dystrophin, Fibrosis, Gene therapy, Inflammation, Muscle",
author = "Gonzalo Cordova and Elisa Negroni and Claudio Cabello-Verrugio and Vincent Mouly and Capucine Trollet",
year = "2018",
month = "4",
day = "10",
doi = "10.3389/fgene.2018.00114",
language = "English",
volume = "9",
journal = "Frontiers in Genetics",
issn = "1664-8021",
publisher = "Frontiers Media S.A.",
number = "APR",

}

Combined therapies for Duchenne muscular dystrophy to optimize treatment efficacy. / Cordova, Gonzalo; Negroni, Elisa; Cabello-Verrugio, Claudio; Mouly, Vincent; Trollet, Capucine.

En: Frontiers in Genetics, Vol. 9, N.º APR, 114, 10.04.2018.

Resultado de la investigación: Short survey

TY - JOUR

T1 - Combined therapies for Duchenne muscular dystrophy to optimize treatment efficacy

AU - Cordova, Gonzalo

AU - Negroni, Elisa

AU - Cabello-Verrugio, Claudio

AU - Mouly, Vincent

AU - Trollet, Capucine

PY - 2018/4/10

Y1 - 2018/4/10

N2 - Duchene Muscular Dystrophy (DMD) is the most frequent muscular dystrophy and one of the most severe due to the absence of the dystrophin protein. Typical pathological features include muscle weakness, muscle wasting, degeneration, and inflammation. At advanced stages DMD muscles present exacerbated extracellular matrix and fat accumulation. Recent progress in therapeutic approaches has allowed new strategies to be investigated, including pharmacological, gene-based and cell-based therapies. Gene and cell-based therapies are still limited by poor targeting and low efficiency in fibrotic dystrophic muscle, therefore it is increasingly evident that future treatments will have to include "combined therapies" to reach maximal efficiency. The scope of this mini-review is to provide an overview of the current literature on such combined therapies for DMD. By "combined therapies" we mean those that include both a therapy to correct the genetic defect and an additional one to address one of the secondary pathological features of the disease. In this mini-review, we will not provide a comprehensive view of the literature on therapies for DMD, since many such reviews already exist, but we will focus on the characteristics, efficiency, and potential of such combined therapeutic strategies that have been described so far for DMD.

AB - Duchene Muscular Dystrophy (DMD) is the most frequent muscular dystrophy and one of the most severe due to the absence of the dystrophin protein. Typical pathological features include muscle weakness, muscle wasting, degeneration, and inflammation. At advanced stages DMD muscles present exacerbated extracellular matrix and fat accumulation. Recent progress in therapeutic approaches has allowed new strategies to be investigated, including pharmacological, gene-based and cell-based therapies. Gene and cell-based therapies are still limited by poor targeting and low efficiency in fibrotic dystrophic muscle, therefore it is increasingly evident that future treatments will have to include "combined therapies" to reach maximal efficiency. The scope of this mini-review is to provide an overview of the current literature on such combined therapies for DMD. By "combined therapies" we mean those that include both a therapy to correct the genetic defect and an additional one to address one of the secondary pathological features of the disease. In this mini-review, we will not provide a comprehensive view of the literature on therapies for DMD, since many such reviews already exist, but we will focus on the characteristics, efficiency, and potential of such combined therapeutic strategies that have been described so far for DMD.

KW - Atrophy

KW - Cell therapy

KW - Duchenne muscular dystrophy

KW - Dystrophin

KW - Fibrosis

KW - Gene therapy

KW - Inflammation

KW - Muscle

UR - http://www.scopus.com/inward/record.url?scp=85045306387&partnerID=8YFLogxK

U2 - 10.3389/fgene.2018.00114

DO - 10.3389/fgene.2018.00114

M3 - Short survey

VL - 9

JO - Frontiers in Genetics

JF - Frontiers in Genetics

SN - 1664-8021

IS - APR

M1 - 114

ER -