Safe harbor loci
AssigneeArsenal Biosciences, Inc.
InventorsXinying Zheng, Brendan Galvin, Somya Khare, Aaron Cooper, Michelle Nguyen, Anzhi Yao
Life of this patent
Estimating the term…
Abstract
Provided herein are safe harbor loci and methods for identifying and using safe harbor loci to engineer cells to express transgenes. The safe harbor loci exhibit increased transgene knock-in efficiency and allow for increased, stable expression of transgenes in engineered cells. Guide ribonucleic acids (gRNAs) may be used for insertion of transgenes in the safe harbor loci.
Claims21
A method of editing a cell having chromosomal DNA, comprising inserting at least one sequence comprising a transgene within a safe harbor locus in the chromosomal DNA of the cell, wherein the safe harbor locus is at any one or more of an sgRNA target loci selected from:
- chr11:128340000-128350000, chr15:92830000-92840000, chr10:33130000-33140000, chr10:72290000-72300000, chr11:65425000-65427000 (NEAT1), chr16:
- 11220000-11230000, chr2:87460000-87470000, chr3:186510000-186520000, chr3:59450000-59460000, chr8:127980000-128000000, chr9:7970000-7980000, APRT, B2M, CAPNS1, CBLB, CD2, CD3E, CD3G, CD5, EDF1, FTL, PTEN, PTPN2, PTPN6, PTPRC, PTPRCAP, RPS23, RTRAF, SERF2, SLC38A1, SMAD2, SOCS1, SRP14, SRSF9, SUB1, TET2, TIGIT, TRAC, or TRIM28.
The method of claim 13, wherein the transgene encodes a protein, a therapeutic agent, a cancer-specific therapeutic agent, a T-cell receptor (TCR), a Prime Receptor (primeR), or a chimeric antigen receptor (CAR).
A guide ribonucleic acid (gRNA) for editing a cell at a safe harbor locus, wherein the gRNA comprises any one of SEQ ID NOS:1-120.
An ex vivo method of obtaining an engineered cell or population thereof, comprising:
- a. obtaining a cell;
- and
- b. genetically modifying the cell by inserting at least one sequence encoding a transgene within a safe harbor locus, wherein the safe harbor locus is at any one or more of an sgRNA target loci selected from:
- chr10:33130000-33140000, chr10:72290000-72300000, chr11:128340000-128350000, chr11:65425000-65427000 (NEAT1), chr15:92830000-92840000, chr16:11220000-11230000, chr2:87460000-87470000, chr3:186510000-186520000, chr3:59450000-59460000, chr8:127980000-128000000, chr9:7970000-7980000, APRT, B2M, CAPNS1, CBLB, CD2, CD3E, CD3G, CD5, EDF1, FTL, PTEN, PTPN2, PTPN6, PTPRC, PTPRCAP, RPS23, RTRAF, SERF2, SLC38A1, SMAD2, SOCS1, SRP14, SRSF9, SUB1, TET2, TIGIT, TRAC, or TRIM28.
Dependencies are inferred from the claim text.