GENOME EDITING USING EFFECTOR OLIGONUCLEOTIDES FOR THERAPEUTIC TREATMENT

摘要

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.

主权项

1. 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.