Patent ID: 11866697
Assignee: THE BROAD INSTITUTE, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A  Y | IPC A  C

Claim 21:
22. An engineered, non-naturally occurring system suitable for modifying an Adenine in a target locus of interest, comprising
a) a guide molecule which comprises a guide sequence linked to a direct repeat sequence, or a nucleotide sequence encoding said guide molecule;
b) a Cpf1 nickase protein, or a nucleotide sequence encoding said Cpf1 nickase protein; and
c) an adenosine deaminase protein or catalytic domain thereof, or a nucleotide sequence encoding said adenosine deaminase protein or catalytic domain thereof;
wherein said adenosine deaminase protein or catalytic domain thereof is covalently or non-covalently linked to said Cpf1 nickase protein or said guide molecule or is adapted to link thereto after delivery;
wherein said guide sequence is capable of hybridizing with a target sequence comprising an Adenine on a first DNA strand at said target locus to form a heteroduplex, wherein said guide sequence comprises a non-pairing Cytosine at a position corresponding to said Adenine resulting in an A-C mismatch in the heteroduplex formed, and wherein said adenosine deaminase protein or catalytic domain thereof deaminates said Adenine in said heteroduplex; and
wherein said Cpf1 nickase protein is capable of nicking a second DNA strand complementary to said first DNA strand.