W. Mark Saltzman,Peter Glazer,Raman Bahal,Nicole Ali McNeer,Danith H. Ly,Elias Quijano
申请号：
US15434978
公开号：
US20170283830A1
申请日:
2017.02.16
申请国别(地区):
US
年份:
2017
代理人:
摘要：
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.
