Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS
Myotonic dystrophies are multisystemic diseases characterized not only by muscle and heart dysfunction but also by CNS alteration. They are now recognized as brain diseases affecting newborns and children for myotonic dystrophy type 1 and adults for both myotonic dystrophy type 1 and type 2. In the...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2017-04-01
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| Series: | Frontiers in Cellular Neuroscience |
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| Online Access: | http://journal.frontiersin.org/article/10.3389/fncel.2017.00101/full |
| _version_ | 1811305052400254976 |
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| author | Genevieve Gourdon Genevieve Gourdon Giovanni Meola |
| author_facet | Genevieve Gourdon Genevieve Gourdon Giovanni Meola |
| author_sort | Genevieve Gourdon |
| collection | DOAJ |
| description | Myotonic dystrophies are multisystemic diseases characterized not only by muscle and heart dysfunction but also by CNS alteration. They are now recognized as brain diseases affecting newborns and children for myotonic dystrophy type 1 and adults for both myotonic dystrophy type 1 and type 2. In the past two decades, much progress has been made in understanding the mechanisms underlying the DM symptoms allowing development of new molecular therapeutic tools with the ultimate aim of curing the disease. This review describes the state of the art for the characterization of CNS related symptoms, the development of molecular strategies to target the CNS as well as the available tools for screening and testing new possible treatments. |
| first_indexed | 2024-04-13T08:18:27Z |
| format | Article |
| id | doaj.art-e463e48a13174d54b1a471fc16afbfb6 |
| institution | Directory Open Access Journal |
| issn | 1662-5102 |
| language | English |
| last_indexed | 2024-04-13T08:18:27Z |
| publishDate | 2017-04-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Cellular Neuroscience |
| spelling | doaj.art-e463e48a13174d54b1a471fc16afbfb62022-12-22T02:54:43ZengFrontiers Media S.A.Frontiers in Cellular Neuroscience1662-51022017-04-011110.3389/fncel.2017.00101256669Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNSGenevieve Gourdon0Genevieve Gourdon1Giovanni Meola2Institut National de la Santé et de la Recherche Médicale UMR1163Paris, FranceLaboratory CTGDM, Institut Imagine, Université Paris Descartes—Sorbonne Paris CitéParis, FranceDepartment of Biomedical Sciences for Health, Policlinico San Donato (IRCCS), University of MilanMilan, ItalyMyotonic dystrophies are multisystemic diseases characterized not only by muscle and heart dysfunction but also by CNS alteration. They are now recognized as brain diseases affecting newborns and children for myotonic dystrophy type 1 and adults for both myotonic dystrophy type 1 and type 2. In the past two decades, much progress has been made in understanding the mechanisms underlying the DM symptoms allowing development of new molecular therapeutic tools with the ultimate aim of curing the disease. This review describes the state of the art for the characterization of CNS related symptoms, the development of molecular strategies to target the CNS as well as the available tools for screening and testing new possible treatments.http://journal.frontiersin.org/article/10.3389/fncel.2017.00101/fullmyotonic dystrophytrinucleotide repeat diseasesDM CNS symptomstherapeutic strategiesanimal models |
| spellingShingle | Genevieve Gourdon Genevieve Gourdon Giovanni Meola Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS Frontiers in Cellular Neuroscience myotonic dystrophy trinucleotide repeat diseases DM CNS symptoms therapeutic strategies animal models |
| title | Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS |
| title_full | Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS |
| title_fullStr | Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS |
| title_full_unstemmed | Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS |
| title_short | Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS |
| title_sort | myotonic dystrophies state of the art of new therapeutic developments for the cns |
| topic | myotonic dystrophy trinucleotide repeat diseases DM CNS symptoms therapeutic strategies animal models |
| url | http://journal.frontiersin.org/article/10.3389/fncel.2017.00101/full |
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