Nonviral vectors for delivering polynucleotides
AssigneeGencia Corporation
InventorShaharyar Khan
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Abstract
Methods and compositions for delivering polynucleotides are provided. One embodiment provides a non-viral vector comprising a recombinant polynucleotide-binding protein comprising a protein transduction domain operably linked to a targeting signal. Methods for modifying the genome of non-nuclear organelles are also provided.
Claims4
A method of compensating for a Leber's Hereditary Optic Neuropathy (LHON) mutation wherein the method comprises directly contacting an eye of a subject with the LHON mutation with a mitochondrial replicon comprising an expression regulatory element operably linked to the LHON wildtype mtDNA sequence and an effective amount of a non-viral vector to package the replicon wherein the non-viral vector comprises a protein transduction domain, a mitochondrial localization signal, and mature transcription factor A, mitochondrial (TFAM).
A method for introducing a polynucleotide into mitochondria, the method comprising contacting one or more cells comprising the mitochondria with the polynucleotide and an effective amount of a fusion protein to package the polynucleotide,
- wherein the fusion protein comprises a protein transduction domain, a mitochondrial localization signal, and a mature transcription factor A, mitochondria (TFAM),
- and delivers the polynucleotide to the mitochondria.
Dependencies are inferred from the claim text.