Muscle‐specific gene editing improves molecular and phenotypic defects in a mouse model of myotonic...
Muscle‐specific gene editing improves molecular and phenotypic defects in a mouse model of myotonic dystrophy type 1
About this item
Full title
Author / Creator
Izzo, Mariapaola , Battistini, Jonathan , Golini, Elisabetta , Voellenkle, Christine , Provenzano, Claudia , Orsini, Tiziana , Strimpakos, Georgios , Scavizzi, Ferdinando , Raspa, Marcello , Baci, Denisa , Frolova, Svetlana , Tastsoglou, Spyros , Zaccagnini, Germana , Garcia‐Manteiga, Jose Manuel , Gourdon, Genevieve , Mandillo, Silvia , Cardinali, Beatrice , Martelli, Fabio and Falcone, Germana
Publisher
United States: John Wiley & Sons, Inc
Journal title
Language
English
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Publication information
Publisher
United States: John Wiley & Sons, Inc
Subjects
More information
Scope and Contents
Contents
Background
Myotonic dystrophy type 1 (DM1) is a genetic multisystemic disease, characterised by pleiotropic symptoms that exhibit notable variability in severity, nature and age of onset. The genetic cause of DM1 is the expansion of unstable CTG‐repeats in the 3′ untranslated region (UTR) of the DMPK gene, resulting in the accumulation of toxic...
Alternative Titles
Full title
Muscle‐specific gene editing improves molecular and phenotypic defects in a mouse model of myotonic dystrophy type 1
Authors, Artists and Contributors
Author / Creator
Battistini, Jonathan
Golini, Elisabetta
Voellenkle, Christine
Provenzano, Claudia
Orsini, Tiziana
Strimpakos, Georgios
Scavizzi, Ferdinando
Raspa, Marcello
Baci, Denisa
Frolova, Svetlana
Tastsoglou, Spyros
Zaccagnini, Germana
Garcia‐Manteiga, Jose Manuel
Gourdon, Genevieve
Mandillo, Silvia
Cardinali, Beatrice
Martelli, Fabio
Falcone, Germana
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_93bb18d30e7d4e9b8459dde9f5c067fe
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_93bb18d30e7d4e9b8459dde9f5c067fe
Other Identifiers
ISSN
2001-1326
E-ISSN
2001-1326
DOI
10.1002/ctm2.70227
