COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO DYSTROPHIA MYOTONICA PROTEIN KINASE

The disclosure relates to compositions and methods for modulating, e.g., reducing or eliminating, the expression of mutated DMPK via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed...

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Bibliographische Detailangaben
Hauptverfasser:	LI, Jiangyu, MOYER, Tyler Christopher, NONNENMACHER, Mathieu Emmanuel, LAKS, Dan Richard
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	The disclosure relates to compositions and methods for modulating, e.g., reducing or eliminating, the expression of mutated DMPK via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having myotonic dystrophy type 1 or another DMPK-related disorder. L'invention concerne des compositions et des procédés de modulation, par exemple, de réduction ou d'élimination, de l'expression de DMPK mutée par administration à l'aide d'un variant de capside de virus adéno-associé (AAV). Les compositions et les procédés de la présente invention sont utiles dans le traitement de sujets chez qui on a diagnostiqué un type de dystrophie myotonique 1 ou un autre trouble lié au DMPK ou suspecté de présenter ceux-ci.