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US 12,496,357 B2Granted
Compositions and methods for treating sickle cell disease
AssigneeYale University
InventorsPeter Glazer, Elias Quijano, W. Mark Saltzman, Hee Won Suh
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Abstract
Peptide nucleic acid (PNA) oligomers that target the β-globin gene and can increase the frequency of recombination of donor oligonucleotide at the site of a Sickle Cell Disease mutation are provided. Nanoparticle formulations for delivering the PNA oligomers and donor oligonucleotides, and potentiating agents for increase the frequency of recombination of the donor oligonucleotide are also provided. Methods of using the PNA oligomers, donor oligonucleotides, nanoparticles, and potentiating agents for treating Sickle Cell Disease are also provided.
Claims19
A nanoparticle comprising
- a core comprising poly(lactic acid) (PLA) comprising a number average molecular weight (Mn) of about 12 kDa;
- a shell comprising a hyperbranched polyglycerol (HPG) comprising a number average molecular weight (Mn) of about 5 kDa;
- and
- a plurality of a peptide nucleic acid (PNA) oligomer entrapped or encapsulated therein, the PNA oligomer comprising
- a Hoogsteen binding peptide nucleic acid 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 genomic DNA and induce strand invasion, displacement, and formation of a triple-stranded molecule among the two PNA segments and the target region.
3 more elements
Dependencies are inferred from the claim text.