THERAPEUTIC ADENO-ASSOCIATED VIRUS DELIVERY OF FUKUTIN RELATED PROTEIN (FKRP) FOR TREATING DYSTROGLYCANOPATHY. DISORDERS INCLUDING LIMB GIRDLE 21 (LGMD21)

Disclosed herein are various optimized nucleic acids encoding the fukutin-related protein (FKRP). Recombinant vectors comprising the optimized nucleic acid (e.g. operatively linked to a muscle specific promoter), such as recombinant adeno-associated virus vectors, for expressing the protein (e.g. in...

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Bibliographische Detailangaben
Hauptverfasser:	Nguyen, Anh, Childers, Martin K, Cooper, Sinclair, Yanez-Cuna, Jorge Omar, Evripioti, Antonia, Low, SiewHui, Iglesias Gonzalez, Juan Manuel, Tretiakova, Anna, Suarez, Lester, Roberts, Michael L, Xiao, Xiao
Format:	Patent
Sprache:	eng
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	Disclosed herein are various optimized nucleic acids encoding the fukutin-related protein (FKRP). Recombinant vectors comprising the optimized nucleic acid (e.g. operatively linked to a muscle specific promoter), such as recombinant adeno-associated virus vectors, for expressing the protein (e.g. in skeletal and cardiac muscle), and therapeutic compositions contains the vectors are also disclosed. Therapeutic methods of administration of the vectors to a subject for the treatment of a subject with a dystroglycanopathy disorder (e.g., limb-girdle muscular dystrophy 2I) are also disclosed.