| abstract |
Methods of transfecting cells in vivo, by administering an injectable pharmaceutical composition including a genome editing composition and a pharmaceutically acceptable carrier to a subject by hydrodynamic injection into a vessel of the subject are disclosed. Typically, the pharmaceutical composition is administered in a volume and at rate of injection suitable to transfect target eukaryotic cells in the subject with an effective amount of the genome editing composition to alter the genome of the target cells. In preferred embodiments the subject is a mammal, such as rodent, or a primate such as a human. The methods can be used to treat one or more symptoms of a genetic disease or condition. |