RECOMBINANT AAV FOR THE GENE THERAPY OF SMA DISEASE

The present invention provides a polynucleotide and recombinant AAV encoding hSMN1. The present invention also provides a method of treating SMA disease comprising administering the recombinant AAV to a subject in need thereof. La présente invention concerne un polynucléotide et un VAA recombinant c...

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Bibliographic Details
Main Authors HOU, Jay, ZHU, Wendy, HUANG, Carol, ZHAO, Lingling, WANG, Ruojie, WU, Yu, SHU, Yanqun
Format Patent
LanguageEnglish
French
Published 28.12.2023
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:The present invention provides a polynucleotide and recombinant AAV encoding hSMN1. The present invention also provides a method of treating SMA disease comprising administering the recombinant AAV to a subject in need thereof. La présente invention concerne un polynucléotide et un VAA recombinant codant pour hSMN1. La présente invention concerne également une méthode de traitement de la maladie SMA, comprenant l'administration du VAA recombinant à un sujet en ayant besoin.
Bibliography:Application Number: WO2023CN101244