IP Library Granted Patent US 8,507,277
Granted Patent B2
US 8,507,277 · App. 11/389,432 · Granted Aug 13, 2013

Nonviral vectors for delivering polynucleotides

Inventor: Shaharyar Khan (Charlottesville, VA)
Assignee: Gencia Corporation
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Quick Facts
Patent No.
US 8,507,277
App. No.
11/389,432
Granted
Aug 13, 2013
Kind
B2
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.

Claims (6)

1. 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).

2. 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.

3. The method of claim 2 wherein the polynucleotide encodes a functional protein operatively linked to an expression control sequence.

4. The method of claim 2 wherein the polynucleotide is a complete mitochondrial genome.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 27, 2006
From: KHAN, SHAHARYAR
To: GENCIA CORPORATION
Reel/Frame 018315/0045 →
Continuity (4)
Continuation In Part 10972963 · Oct 25, 2004
Provisional Application 60568436 · May 5, 2004
Provisional Application 60513983 · Oct 24, 2003
Related Publication 20060211647A1 · Sep 21, 2006