Compositions And Methods For Treatment Of Cystic Fibrosis


Ontology type: sgo:Patent     


Patent Info

DATE

N/A

AUTHORS

GLAZER, PETER , SALTZMAN, W. MARK , EGAN, MARIE , MCNEER, Nicole Ali

ABSTRACT

Compositions and methods of genome engineering in vitro and in vivo are provided. In some embodiments, the compositions are triplex forming molecules that bind or hybridize to a target region sequence in the human cystic fibrosis transmembrane conductance regulator (CFTR) gene. Preferably the triplex forming molecules are peptide nucleic acids that include a Hoogsteen binding peptide nucleic acid (PNA) segment and a Watson-Crick binding PNA segment collectively totaling no more than 50 nucleobases in length, wherein the two segments can bind or hybridize to a target region in the CFTR gene having a polypurine sequences and induce strand invasion, displacement, and formation of a triple-stranded molecule among the two PNA segments and the target region's sequence. Methods of using the triplex forming molecules to treat cystic fibrosis are also provided. More... »

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