VIRAL VECTOR FOR COMBINATION THERAPY

The invention described herein provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, that co-express two or more GOIs. The vectors of the invention can be broadly used to treat a number of genetic disorders such as trinucleotide repeat expansion disorders.

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Bibliographic Details
Main Authors Ozsolak, Fatih, Mandava, Sarath Chandra, Reyes, Eric, Schneider, Joel, Soustek-Kramer, Meghan S
Format Patent
LanguageEnglish
Published 15.06.2023
Subjects
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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Summary:The invention described herein provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, that co-express two or more GOIs. The vectors of the invention can be broadly used to treat a number of genetic disorders such as trinucleotide repeat expansion disorders.
Bibliography:Application Number: US202117791840