Transposition-based therapies
AssigneeSaliogen Therapeutics, Inc.
InventorsJoseph J. Higgins, Scott McMillan, Ray Tabibiazar
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Abstract
Gene therapy compositions and methods are provided that make use of novel transposases and/or chimeric transposases for targeted transposition.
Claims28
A method for inserting a gene into the genome of a cell, comprising contacting the cell with a composition comprising a nucleic acid encoding a transposase enzyme, wherein the transposase enzyme comprises an amino acid sequence having at least 95% sequence identity to SEQ ID NO:
- 2 and has a non-polar aliphatic amino acid at position 2 of SEQ ID NO:
- 2,
- wherein the transposase enzyme further comprises a substitution C13X2, wherein X2 is selected from lysine (K), arginine (R), and histidine (H).
A method for inserting a gene into the genome of a cell, comprising contacting the cell with a composition comprising a nucleic acid encoding a transposase enzyme,
- wherein the transposase enzyme comprises an amino acid sequence having at least 95% sequence identity to SEQ ID NO:
- 2 and has a non-polar aliphatic amino acid at position 2 of SEQ ID NO:
- 2, wherein the transposase enzyme further comprises a substitution N125X3, wherein X3 is selected from is selected from lysine (K), arginine (R), and histidine (H).
A method for inserting a gene into the genome of a cell, comprising contacting the cell with a composition comprising a nucleic acid encoding a transposase enzyme,
- wherein the transposase enzyme comprises an amino acid sequence having at least 95% sequence identity to SEQ ID NO:
- 2 and has a non-polar aliphatic amino acid at position 2 of SEQ ID NO:
- 2, wherein the transposase enzyme further comprises two or more of substitutions S8P, C13R, and N125K.
A method for inserting a gene into the genome of a cell, comprising contacting the cell with a composition comprising a first nucleic acid encoding a transposase enzyme and a DNA binding domain and a second nucleic acid encoding a transposon,
- wherein the transposase enzyme comprises an amino acid sequence having at least 97% sequence identity to SEQ ID NO:
- 2 and comprises two or more of substitutions of S8P, C13R, and N125K,
- wherein the transposase enzyme is operatively linked to the DNA binding domain, and
- wherein the first nucleic acid and second nucleic acid are contained in a LNP.
Dependencies are inferred from the claim text.