Base editing of pcsk9 and methods of using same for treatment of disease

A composition comprises a protein having a programmable DNA binding domain (e.g. an inactive Cas9 nuclease or nickase) or a nucleic acid encoding that protein and a guide polynucleotide comprising a tracr sequence having at least 80% sequence identity to SEQ ID NO:61 and a spacer sequence which corr...

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Bibliographic Details
Main Authors Kallanthottathil G Rajeev, Alexandra Chadwick, Christopher Cheng, Ellen Rohde, Caroline Reiss
Format Patent
LanguageEnglish
Published 03.07.2024
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:A composition comprises a protein having a programmable DNA binding domain (e.g. an inactive Cas9 nuclease or nickase) or a nucleic acid encoding that protein and a guide polynucleotide comprising a tracr sequence having at least 80% sequence identity to SEQ ID NO:61 and a spacer sequence which corresponds to a protospacer on a gene encoding PCSK1 (proprotein convertase subtilisin-kexin type 9). In use, this composition may edit the PCSK9 gene. The composition may be formulated as a pharmaceutical for use in treating atherosclerosis. A polynucleotide of SEQ ID NO: 2192 encoding a base editing protein is also disclosed. Also disclosed are methods to edit the ANGPTL3 gene.
Bibliography:Application Number: GB20230017179