MORPHOLINO TARGETING DUX4 FOR TREATING FSHD

A composition and method is provided that comprises a morpholino oligonucleotide capable of inhibiting DUX4 expression. The morpholino oligonucleotide can provide a therapeutic effect in individuals with facioscapulohumeral muscular dystrophy. Further, the composition and method may comprise a dendr...

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Bibliographic Details
Main Authors MULTHAUP, MEGAN, KYBA, MICHAEL
Format Patent
LanguageEnglish
French
Published 30.07.2015
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
DERIVATIVES THEREOF
ENZYMOLOGY
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
NUCLEIC ACIDS
NUCLEOSIDES
NUCLEOTIDES
ORGANIC CHEMISTRY
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
SUGARS
VINEGAR
WINE
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Summary:A composition and method is provided that comprises a morpholino oligonucleotide capable of inhibiting DUX4 expression. The morpholino oligonucleotide can provide a therapeutic effect in individuals with facioscapulohumeral muscular dystrophy. Further, the composition and method may comprise a dendrimer delivery moiety. La présente invention concerne une composition et un procédé comprenant un oligonucléotide morpholino apte à inhiber l'expression du DUX4. Ledit oligonucléotide morpholino peut procurer un effet thérapeutique chez des individus atteints de dystrophie musculaire facio-scapulo-humérale. En outre, ladite composition et ledit procédé peuvent comprendre une fraction d'administration de dendrimère.
Bibliography:Application Number: WO2013US25461