Genome editing using effector oligonucleotides for therapeutic treatment
AssigneeResearch Foundation to Cure AIDS, Inc.
InventorKambiz SHEKDAR
Life of this patent
Estimating the term…
Abstract
The invention provides compositions and methods of making and using effector oligonucleotides, including effector oligonucleotides with greater than one mismatch as compared to its target sequence. These effector oligonucleotides are useful for improving the efficiency of genomic editing as well as providing therapeutic benefits to individuals in need thereof.
Claims53
A method of making recombinant cells comprising contacting cells comprising a target sequence within a gene to be altered with an effector oligonucleotide targeted to the target sequence and comprising more than one mismatch as compared to the target sequence;
- and allowing the effector oligonucleotide to alter the target sequence in the cells.
A composition comprising a recombinant cell comprising an altered target sequence and an effector oligonucleotide targeted to the target sequence.
A composition comprising a substantially enriched population of recombinant stem cells, wherein the recombinant stem cells comprise a Δ32 deletion in a CCR5 gene.
An effector oligonucleotide having a sequence selected from the group consisting of SEQ ID NO:5, SEQ ID NO:6, SEQ ID NO:7, SEQ ID NO:8, SEQ ID NO:9, SEQ ID NO:10, SEQ ID NO:11, SEQ ID NO:
- 12, SEQ ID NO:13, SEQ ID NO:14, SEQ ID NO:
- 15, SEQ ID NO:16, SEQ ID NO:
- 17, SEQ ID NO:18, SEQ ID NO:
- 19, SEQ ID NO:20, SEQ ID NO:21, SEQ ID NO:22, SEQ ID NO:23, SEQ ID NO:24, SEQ ID NO:25, SEQ ID NO:26, SEQ ID NO:27, SEQ ID NO:28, SEQ ID NO:29, SEQ ID NO:30, SEQ ID NO:31, SEQ ID NO:32, SEQ ID NO:33, SEQ ID NO:34, SEQ ID NO:35, SEQ ID NO:36, and SEQ ID NO:37.
Dependencies are inferred from the claim text.