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公开(公告)号:US20190233816A1
公开(公告)日:2019-08-01
申请号:US16256003
申请日:2019-01-24
Applicant: Massachusetts Institute of Technology
Inventor: Robert Samuel LANGER , Hao Yin , Daniel G. Anderson , Wen Xue , Chun-Qing Song
IPC: C12N15/113 , A61K31/7105 , C12N15/85 , C12N15/90 , C12N9/22
CPC classification number: C12N15/113 , A61K31/7105 , C12N9/22 , C12N15/111 , C12N15/85 , C12N15/907 , C12N2310/20 , C12N2310/343 , C12N2320/53 , C12Y301/11002 , C12N2310/322 , C12N2310/3531
Abstract: The disclosure relates to compositions comprising and methods for chemical modification of single guide RNA (sgRNA), tracrRNA and/or crRNA used individually or in combination with one another or Cas system components. Compositions comprising modified ribonucleic acids have been designed with chemical modification for even higher efficiency as unmodified native strand of sgRNA. Administration of modified ribonucleic acids will allow decreased immune response when administered to a subject, increased stability, increased editing efficiency and facilitated in vivo delivery of sgRNA via various delivery platforms. The disclosure also relates to methods of decreasing off-target effect of CRISPR and a CRISPR complex.