Gene Therapy Using Transposon-Based Vectors
AssigneesThe Board of Supervisors of Louisiana State University and Agricultural and Mechanical, College, TransGenRx, Inc.
InventorsRichard Cooper, Frederick M. Enright, William C. Fioretti
Life of this patent
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Abstract
Methods and compositions are presented for the administration of transposon-based vectors to an animal or human to provide gene therapy to the animal or human
Claims24
1-7. (canceled)
A mammal, bird, or a human comprising an isolated polynucleotide sequence. comprising:
- a) one or more genes of interest operably-linked to one or more promoters;
- b) a poly A nucleotide sequence located 3′ to the one or more genes of interest;
- and,
- c) transposase insertion sequences recognized by a bacterial transposase, wherein the one or more genes of interest and their operably-linked promoters are flanked by the transposase insertion sequences and the one or more additional promoters are cell-specific promoters or constitutive promoters.
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A cell of the mammal, bird, or human of claim 6 8.
A method of providing gene therapy to an animal or a human comprising administering to the animal or the human a transposon-based vector comprising an isolated polynucleotide sequence encoding:
- a) a gene operably linked to a first promoter, the gene encoding for a transposase;
- and,
- b) one or more genes of interest operably-linked to one or more additional promoters, wherein the one or more genes of interest and their operably-linked promoters are flanked by transposase insertion sequences recognized by the transposase, and wherein the first promoter and the one or more additional promoters are cell-s ecific promoters or constitutive promoters.
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Dependencies are inferred from the claim text.