TRIPLE FUNCTION ADENO-ASSOCIATED VIRUS (AAV) VECTORS FOR THE TREATMENT OF C9ORF72 ASSOCIATED DISEASES

TRIPLE FUNCTION ADENO-ASSOCIATED VIRUS (AAV) VECTORS FOR THE TREATMENT OF C9ORF72 ASSOCIATED DISEASES

The present disclosure provides isolated promoters, transgene expression cassettes, vectors, kits, and methods for treatment of C9ORF72 associated diseases, including ALS and FTD.

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Bibliographische Detailangaben
Hauptverfasser: ZHU, Peixin, WANG, Xijia, PENNOCK, Steven, SHEARMAN, Mark
Format: Patent
Sprache:eng ; fre ; ger
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 present disclosure provides isolated promoters, transgene expression cassettes, vectors, kits, and methods for treatment of C9ORF72 associated diseases, including ALS and FTD.