公开(公告)号：EP3416976A2

公开(公告)日：2018-12-26

申请号：EP17711024.4

申请日：2017-02-16

申请人： Yale University ,  Carnegie Mellon University

发明人： SALTZMAN, W., Mark ,  GLAZER, Peter ,  BAHAL, Raman ,  MCNEER, Nicole, Ali ,  LY, Danith, H. ,  QUIJANO, Elias

IPC分类号： C07K14/00

CPC分类号： C12N15/907 ,  A61K9/1647 ,  A61K48/005 ,  C07K14/003 ,  C12N15/102 ,  C12N15/11 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/15 ,  C12N2310/152 ,  C12N2310/318 ,  C12N2310/3181 ,  C12N2310/334 ,  C12N2310/351 ,  C12N2310/3519

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.

公开(公告)号：EP3728206A1

公开(公告)日：2020-10-28

申请号：EP18891446.9

申请日：2018-12-21

申请人： CARNEGIE MELLON UNIVERSITY

发明人： LY, Danith, H. ,  HSIEH, Wei-Che ,  BAHAL, Raman

IPC分类号： C07D239/47 ,  C07D239/54 ,  C07D251/16

公开(公告)号：EP3844275A1

公开(公告)日：2021-07-07

申请号：EP19768978.9

申请日：2019-08-30

申请人： Yale University

发明人： QUIJANO, Elias ,  RICCIARDI, Adele ,  BAHAL, Raman ,  TURCHICK, Audrey ,  ECONOMOS, Nicholas ,  SALTZMAN, W. Mark ,  GLAZER, Peter

IPC分类号： C12N15/113 ,  C12N15/85 ,  A61K39/00 ,  A61K48/00 ,  C12N15/10 ,  C12N15/90