Compositions and methods for modifying a predetermined target nucleic acid sequence
AssigneeTargetgene Biotechnologies Ltd.
InventorsYoel Moshe Shiboleth, Dan Michael Weinthal
Life of this patent
Estimating the term…
Abstract
Provided herein are compositions and methods for modifying a predetermined nucleic acid sequence. A programmable nucleoprotein molecular complex containing a polypeptide moiety and a specificity conferring nucleic acid (SCNA) which assembles in-vivo, in a target cell, and is capable of interacting with the predetermined target nucleic acid sequence is provided. The programmable nucleoprotein molecular complex is capable of specifically modifying and/or editing a target site within the target nucleic acid sequence and/or modifying the function of the target nucleic acid sequence.
Claims17
A synthetic specificity conferring nucleic acid (SCNA) comprising:
- (a) a specificity-defining region comprising a nucleotide sequence complementary to a target region of a target DNA molecule,
- (b) a spacer comprising between 1 nucleotide and 6 nucleotides positioned between the specificity-defining region and a recognition region, and
- (c) the recognition region, wherein the recognition region is heterologous to the specificity-defining region and capable of specifically interacting with a polypeptide, wherein the SCNA and the polypeptide are capable of forming a nucleoprotein complex that interacts with the target DNA molecule, wherein the SCNA provides the specificity and binding capability of the nucleoprotein complex to a predetermined target site within the target DNA molecule, wherein the SCNA comprises an RNA molecule, and wherein the polypeptide does not comprise a recombinase domain.
A synthetic specificity conferring nucleic acid (SCNA) comprising:
- (a) a specificity-defining region comprising a nucleotide sequence complementary to a target region of a target DNA molecule,
- (b) a spacer comprising between 1 nucleotide and 6 nucleotides positioned between the specificity-defining region and a recognition region, and
- (c) the recognition region, wherein the recognition region is separate from the specificity-defining region and capable of specifically interacting with a polypeptide, wherein the SCNA and the polypeptide are capable of forming a nucleoprotein complex, and wherein the SCNA is capable of guiding the nucleoprotein complex to the target region of the target DNA molecule, wherein the nucleoprotein complex interacts with the target DNA molecule, wherein the SCNA provides the specificity and binding capability of the nucleoprotein complex to a predetermined target site within the target DNA molecule, and wherein the polypeptide does not comprise a recombinase domain.
Dependencies are inferred from the claim text.