Genetic correction of mutated genes
AssigneeDuke University
InventorsCharles Gersbach, David Ousterout
Life of this patent
Estimating the term…
Abstract
Disclosed herein are transcription activator-like effector nuclease (TALEN)-related compositions and methods of using said TALENs for correcting mutant genes.
Claims15
An isolated polynucleotide comprising a nucleotide sequence encoding an engineered transcription activator-like effector nuclease (TALEN) protein that binds to a dystrophin gene, wherein the TALEN protein binds upstream from a premature stop codon on the dystrophin gene, downstream from a premature stop codon on the dystrophin gene, a region in exon 51 of the dystrophin gene, or a region in the 5′ UTR of the dystrophin gene, and wherein the TALEN protein comprises an amino acid sequence selected from the group consisting of SEQ ID NO:
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Dependencies are inferred from the claim text.