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Abstract

Embodiments of the present invention relate to methods and compositions for tissue- specific delivery of a gene therapeutic, transgenic nucleic acid in mammals. Methods and compositions of the invention include the steps of providing a nucleic acid comprising a transgene flanked by two terminal repeats and, within the same or on a separate nucleic acid, a nucleotide sequence encoding a transposase, wherein the transgene comprises a biotherapeutic gene, contacting the nucleic acid with perfluorocarbon gas-filled microbubbles to form a mixture, introducing the mixture into the bloodstream of a mammal, and focusing ultrasound pulses on a specific tissue of said mammal, wherein said pulses disrupt said microbubbles of said mixture and release said nucleic acid into the bloodstream within the target tissue, thereby enabling uptake of the transgenic nucleic acid into the cells of said target tissue.

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First Claim