公开(公告)号：US09925248B2

公开(公告)日：2018-03-27

申请号：US14838057

申请日：2014-08-29

Applicant: TEMPLE UNIVERSITY OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION

Inventor： Kamel Khalili ,  Wenhui Hu

IPC: A61K48/00 ,  A61K38/46 ,  A61K9/00 ,  A61K35/12 ,  A61K45/06 ,  C12N7/00 ,  C12N9/22 ,  C12N15/11 ,  A61K39/21 ,  C12N15/55 ,  C12N15/79 ,  C12N15/85 ,  C12N15/86

CPC classification number: A61K38/465 ,  A61K9/0034 ,  A61K35/12 ,  A61K45/06 ,  A61K48/00 ,  A61K48/005 ,  C12N7/00 ,  C12N9/22 ,  C12N15/111 ,  C12N2310/20 ,  C12N2320/30 ,  C12N2740/16063 ,  C12Y301/21

Abstract: A method of inactivating a proviral DNA integrated into the genome of a host cell latently infected with a retrovirus by treating the host cell with a composition comprising a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, and two or more different guide RNAs (gRNAs), wherein each of the at least two gRNAs is complementary to a different target nucleic acid sequence in a long terminal repeat (LTR) in the proviral DNA, and inactivating the proviral DNA. A composition for use in inactivating a proviral DNA integrated into the genome of a host cell latently infected with a retrovirus including isolated nucleic acid sequences comprising a CRISPR-associated endonuclease and a guide RNA, wherein the guide RNA is complementary to a target sequence in a human immunodeficiency virus.

公开(公告)号：US20180073019A1

公开(公告)日：2018-03-15

申请号：US15559902

申请日：2016-03-18

Applicant: Temple University - of the Commonwealth System of Higher Education

Inventor： Kamel Khalili

IPC: C12N15/11 ,  C12N9/22 ,  A61K48/00 ,  C12N15/86 ,  A61K45/06 ,  C12N7/00

CPC classification number: C12N15/111 ,  A61K45/06 ,  A61K48/005 ,  A61K48/0075 ,  C12N7/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/1132 ,  C12N15/86 ,  C12N2310/20 ,  C12N2310/3519 ,  C12N2320/30 ,  C12N2740/16062 ,  C12N2800/107 ,  C12N2800/22 ,  C12N2830/00 ,  C12N2830/30 ,  C12N2830/60

Abstract: Compositions and methods are provided for Tat-inducible expression of a CRISPR-associated endonuclease by a truncated HIV LTR promoter containing at least a core region and a TAR region of a HIV LTR promoter. The compositions may be used as a therapeutic treatment for the treatment and/or prevention of HIV.

公开(公告)号：US20160017301A1

公开(公告)日：2016-01-21

申请号：US14838057

申请日：2014-08-29

Applicant: TEMPLE UNIVERSITY OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION

Inventor： Kamel Khalili ,  Wenhui Hu

IPC: C12N9/22 ,  C12N7/00 ,  A61K48/00 ,  A61K35/12 ,  A61K9/00 ,  A61K45/06

CPC classification number: A61K38/465 ,  A61K9/0034 ,  A61K35/12 ,  A61K45/06 ,  A61K48/00 ,  A61K48/005 ,  C12N7/00 ,  C12N9/22 ,  C12N15/111 ,  C12N2310/20 ,  C12N2320/30 ,  C12N2740/16063 ,  C12Y301/21

Abstract: A method of inactivating a proviral DNA integrated into the genome of a host cell latently infected with a retrovirus by treating the host cell with a composition comprising a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, and two or more different guide RNAs (gRNAs), wherein each of the at least two gRNAs is complementary to a different target nucleic acid sequence in a long terminal repeat (LTR) in the proviral DNA, and inactivating the proviral DNA. A composition for use in inactivating a proviral DNA integrated into the genome of a host cell latently infected with a retrovirus including isolated nucleic acid sequences comprising a CRISPR-associated endonuclease and a guide RNA, wherein the guide RNA is complementary to a target sequence in a human immunodeficiency virus.

Abstract translation: 通过用包含聚集的定期间隔短回文序列（CRISPR）相关内切核酸酶的组合物和两种或更多种不同的引导RNA来处理宿主细胞，使整合到潜伏感染逆转录病毒的宿主细胞基因组中的前病毒DNA失活的方法 （gRNA），其中所述至少两个gRNA中的每一个与前病毒DNA中的长末端重复（LTR）中的不同靶核酸序列互补，并使前病毒DNA失活。 用于灭活整合到潜伏感染逆转录病毒的宿主细胞的基因组中的前病毒DNA的组合物，包括分离的包含CRISPR相关内切核酸酶和引导RNA的核酸序列，其中所述指导RNA与 人类免疫缺陷病毒。