公开(公告)号：US20200268907A1

公开(公告)日：2020-08-27

申请号：US16851636

申请日：2020-04-17

申请人： The Johns Hopkins University

发明人： Vinod Jaskula-Ranga ,  Donald Zack

IPC分类号： A61K48/00 ,  C12N15/113 ,  C12N15/63 ,  C12N15/85 ,  C12N15/90 ,  C12N15/11 ,  C12N15/861 ,  C12N15/86

摘要： The presently disclosed subject matter provides compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter. In particular, compositions and methods are provided for the use of the H1 promoter to express CRISPR guide RNA (gRNA) with altered specificity of the 5′ nucleotide, as well as use of the H1 promoter sequence as a bidirectional promoter to express Cas9 nuclease and the gRNA simultaneously. Compositions and methods are also provided for the expression and regulation of gRNA expression in vivo through the use of RNA ribozymes and regulatable aptazymes.

公开(公告)号：US20190365928A1

公开(公告)日：2019-12-05

申请号：US16445352

申请日：2019-06-19

申请人： The Johns Hopkins University

发明人： Vinod Jaskula-Ranga ,  Donald Zack

IPC分类号： A61K48/00 ,  C12N15/113 ,  C12N15/63 ,  C12N15/85 ,  C12N15/90 ,  C12N15/11

摘要： The presently disclosed subject matter provides compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter. In particular, compositions and methods are provided for the use of the H1 promoter to express CRISPR guide RNA (gRNA) with altered specificity of the 5′ nucleotide, as well as use of the H1 promoter sequence as a bidirectional promoter to express Cas9 nuclease and the gRNA simultaneously. Compositions and methods are also provided for the expression and regulation of gRNA expression in vivo through the use of RNA ribozymes and regulatable aptazymes.

公开(公告)号：US10406245B2

公开(公告)日：2019-09-10

申请号：US16011091

申请日：2018-06-18

申请人： The Johns Hopkins University

发明人： Vinod Jaskula-Ranga ,  Donald Zack

IPC分类号： A61K48/00 ,  C12N15/63 ,  C12N15/85 ,  C12N15/90 ,  C12N15/113 ,  C12N15/11 ,  C12N15/861 ,  C12N15/86

摘要： The presently disclosed subject matter provides compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter. In particular, compositions and methods are provided for the use of the H1 promoter to express CRISPR guide RNA (gRNA) with altered specificity of the 5′ nucleotide, as well as use of the H1 promoter sequence as a bidirectional promoter to express Cas9 nuclease and the gRNA simultaneously. Compositions and methods are also provided for the expression and regulation of gRNA expression in vivo through the use of RNA ribozymes and regulatable aptazymes.

公开(公告)号：US20180369420A1

公开(公告)日：2018-12-27

申请号：US16011091

申请日：2018-06-18

申请人： The Johns Hopkins University

发明人： Vinod Jaskula-Ranga ,  Donald Zack

IPC分类号： A61K48/00 ,  C12N15/11 ,  C12N15/113 ,  C12N15/63 ,  C12N15/90 ,  C12N15/85

CPC分类号： A61K48/0066 ,  C07K2319/10 ,  C07K2319/60 ,  C12N15/111 ,  C12N15/1138 ,  C12N15/63 ,  C12N15/85 ,  C12N15/86 ,  C12N15/907 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2330/51 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2830/205 ,  C12N2830/85

摘要： The presently disclosed subject matter provides compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter. In particular, compositions and methods are provided for the use of the H1 promoter to express CRISPR guide RNA (gRNA) with altered specificity of the 5′ nucleotide, as well as use of the H1 promoter sequence as a bidirectional promoter to express Cas9 nuclease and the gRNA simultaneously. Compositions and methods are also provided for the expression and regulation of gRNA expression in vivo through the use of RNA ribozymes and regulatable aptazymes.