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AssigneeScribe Therapeutics Inc.
InventorsAddison WRIGHT, Sean HIGGINS, Maroof ADIL, Cole URNES, Santhosh KARANTH, Brett T. STAAHL, Sarah DENNY, Benjamin OAKES, Oleh KRUPA, Matthew HARMS, Manuel MOHR, Wenyuan ZHOU, Kian TAYLOR
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Provided herein are systems comprising Class 2, Type V CRISPR proteins and guide nucleic acids (gRNA) useful in the modification of a PCSK9 gene. The systems are also useful for introduction into cells, for example eukaryotic cells. Also provided are methods of using such systems to modify cells having such mutations.
A system comprising an engineered CasX protein and a guide nucleic acid (gRNA), wherein the gRNA has a scaffold comprising a sequence selected from the group consisting of SEQ ID NOS:
(canceled)
(canceled)
11-30. (canceled)
32-67. (canceled)
69-76. (canceled)
79-81. (canceled)
83-84. (canceled)
86-89. (canceled)
91-95. (canceled)
A method of modifying a PCSK9 gene in a population of cells in vitro or ex vivo, the method comprising introducing into cells of the population a system comprising an engineered CasX protein and a gRNA, wherein the gRNA has a scaffold comprising a sequence selected from the group consisting of SEQ ID NOS:
97-109. (canceled)
111-127. (canceled)
A system comprising an engineered CasX protein and a gRNA, wherein the engineered CasX protein comprises a target strand loading (TSL) domain comprising the sequence of amino acids 811 to 920 of SEQ ID NO:
Dependencies are inferred from the claim text.