METHODS AND COMPOSITIONS OF OTC CONSTRUCTS AND VECTORS

Provided herein are methods and compositions related to nucleic acids encoding ornithine transcarbamylase (OTC), such as nucleic acids comprising an OTC codon-optimized sequence, as well as related vectors, such as AAV vectors. Also, provided are methods for administering AAV vectors that comprise a...

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Bibliographic Details
Main Authors De Sabbata, Giulia, Kishimoto, Takashi Kei, Muro, Andres, Keller, Peter
Format Patent
LanguageEnglish
Published 06.02.2020
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
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Summary:Provided herein are methods and compositions related to nucleic acids encoding ornithine transcarbamylase (OTC), such as nucleic acids comprising an OTC codon-optimized sequence, as well as related vectors, such as AAV vectors. Also, provided are methods for administering AAV vectors that comprise a sequence that encodes an enzyme associated with an urea cycle disorder and an expression control sequence, in combination with synthetic nanocarriers coupled to an immunosuppressant.
Bibliography:Application Number: US201916513566