BASE EDITING OF PCSK9 AND METHODS OF USING SAME FOR TREATMENT OF DISEASE

Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause...

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Bibliographic Details
Main Authors CHADWICK, ALEXANDRA, CHENG, CHRISTOPHER, RAJEEV, KALLANTHOTTATHIL G, ROHDE, ELLEN, REISS, CAROLINE
Format Patent
LanguageEnglish
French
Published 14.10.2021
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
DERIVATIVES THEREOF
ENZYMOLOGY
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
NUCLEIC ACIDS
NUCLEOSIDES
NUCLEOTIDES
ORGANIC CHEMISTRY
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
SUGARS
VINEGAR
WINE
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Summary:Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed. L'invention concerne des compositions pour la modification ou l'édition de gènes et des procédés d'utilisation de celles-ci pour traiter ou prévenir certaines conditions. L'invention concerne des compositions et des procédés spécifiques capables d'éditer de manière sûre et efficace des cibles géniques exprimées dans le foie pour réduire durablement la LDL-C, ce qui permet de traiter une cause principale de maladie cardiovasculaire.
Bibliography:Application Number: CA20213179863