COMPOSITIONS AND METHODS FOR THE TARGETING OF C9orf72

Provided herein are Class 2 Type V systems comprising nucleases, guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a C9orf72 gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations or duplications...

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Main Authors STAAHL, Brett T, BANEY, Katherine, DENNY, Sarah, URNES, Cole, COLIN, Isabel, SPINNER, Hannah, ADIL, Maroof, TAYLOR, Kian, HIGGINS, Sean, OAKES, Benjamin
Format Patent
LanguageEnglish
Published 19.09.2024
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:Provided herein are Class 2 Type V systems comprising nucleases, guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a C9orf72 gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations or duplications in the C9orf72 gene. Also provided are methods of using such systems to modify cells having such mutations or duplications.
Bibliography:Application Number: US202217932798