Inventors:
- New Haven CT, US
- Pittsburg PA, US
Raman Bahal - Hamden CT, US
Nicole Ali McNeer - Westport CT, US
Danith H. Ly - Pittsburgh PA, US
Elias Quijano - New Haven CT, US
International Classification:
C12N 15/90
A61K 48/00
A61K 9/16
C12N 15/11
Abstract:
Compositions and methods for enhancing targeted gene editing and methods of use thereof are disclosed. In the most preferred embodiments, gene editing is carried out utilizing a gene editing composition such as triplex-forming oligonucleotides, CRISPR, zinc finger nucleases, TALENS, or others, in combination with a gene modification potentiating agent such as stem cell factor (SCF), a CHK1 or ATR inhibitor, or a combination thereof. A particular preferred gene editing composition is triplex-forming peptide nucleic acids (PNAs) substituted at the γ position for increased DNA binding affinity. Nanoparticle compositions for intracellular delivery of the gene editing composition are also provided and particular advantageous for use with in vivo applications.