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公开(公告)号：US20190225963A1
公开(公告)日：2019-07-25
申请号：US15998558
申请日：2017-02-13
Inventor： Kamel KHALILI , Wenhui HU
Abstract: Compositions for the in vivo delivery of a gene editing CRISPR/Cas9 complex was developed to eliminate integrated retroviral DNA sequences from latently infected human cells and animal disease models
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公开(公告)号：US20180200343A1
公开(公告)日：2018-07-19
申请号：US15902263
申请日：2018-02-22
Inventor： Kamel KHALILI , Wenhui HU
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 treating a subject having or at risk for having a virus infection, by administering a therapeutically effective amount of a composition comprising a vector encoding a CRISPR-associated endonuclease and at least two guide RNAs that are complementary to two target sequences spanning from the 5′- to 3′-LTRs of the sequence in the virus, and completely excising a fragment of greater than 9000-bp of integrated proviral DNA that spanned from its 5′- to 3′-LTRs. A method of treating a subject having or at risk for having a genetic caused disease, by administering a therapeutically effective amount of a composition comprising a vector encoding a CRISPR-associated endonuclease and at least two guide RNAs that are complementary to two target sequences spanning from the sequence of the subjects DNA greater than 9000-bp that is chromosomally integrated and causes the genetic caused disease, and excising the chromosomally integrated sequence.
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公开(公告)号：US20180169193A1
公开(公告)日：2018-06-21
申请号：US15879877
申请日：2018-01-25
Inventor： Kamel KHALILI , Wenhui HU
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: Methods of inactivating a proviral DNA genome or a DNA genome 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 multiplex 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) of the proviral DNA that is unique from the genome of the host cell, cleaving a double strand of the proviral DNA at a first target protospacer sequence with the CRISPR-associated endonuclease, cleaving a double strand of the proviral DNA at a second target protospacer sequence with the CRISPR-associated endonuclease, excising an entire proviral genome of the proviral DNA, and eradicating the proviral DNA from the host cell.
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公开(公告)号：US20160250300A1
公开(公告)日：2016-09-01
申请号：US15148261
申请日：2016-05-06
Inventor： Kamel KHALILI , Wenhui HU
IPC: A61K38/46
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.
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公开(公告)号：US20180236043A1
公开(公告)日：2018-08-23
申请号：US15882228
申请日：2018-01-29
Inventor： Kamel KHALILI , Wenhui HU
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 treating a subject having or at risk for having a virus infection, by administering to the subject a therapeutically effective amount of a composition comprising a vector encoding a CRISPR-associated endonuclease and at least two guide RNAs, wherein the guide RNAs are complementary to two target sequences spanning from the 5′- to 3′-LTRs of the sequence in the virus. A method of treating a subject having or at risk for having a virus infection, by administering to the subject a therapeutically effective amount of a composition comprising a vector encoding a CRISPR-associated endonuclease and at least two guide RNAs, wherein the guide RNAs are complementary to two target sequences spanning from the 5′- to 3′-LTRs of the sequence in the virus, and causing neither genotoxicity nor off-target editing to the host.
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Patent Agency Ranking
公开(公告)号：US20180236041A1
公开(公告)日：2018-08-23
申请号：US15882197
申请日：2018-01-29
Inventor： Kamel KHALILI , Wenhui HU
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 treating a subject having or at risk for having a virus infection, by administering a therapeutically effective amount of a composition comprising a vector encoding a CRISPR-associated endonuclease and at least two guide RNAs that are complementary to two target sequences spanning from the 5′- to 3′-LTRs of the sequence in the virus, and completely excising a fragment of greater than 9000-bp of integrated proviral DNA that spanned from its 5′- to 3′-LTRs. A method of treating a subject having or at risk for having a genetic caused disease, by administering a therapeutically effective amount of a composition comprising a vector encoding a CRISPR-associated endonuclease and at least two guide RNAs that are complementary to two target sequences spanning from the sequence of the subjects DNA greater than 9000-bp that is chromosomally integrated and causes the genetic caused disease, and excising the chromosomally integrated sequence.
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公开(公告)号：US20180228875A1
公开(公告)日：2018-08-16
申请号：US15950227
申请日：2018-04-11
Inventor： Kamel Khalili , Wenhui HU
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 personalized method of inactivating a proviral DNA integrated into the genome of a host cell latently infected with a retrovirus, by determining a nucleic acid sequence of the HIV-1 proviral DNA harbored by a subject, designing two or more different multiplex guide RNAs (gRNAs) complementary to the HIV-1 proviral DNA sequences in the subject, administering to the subject a therapeutically effective amount of a composition comprising a CRISPR-associated endonuclease, and the two or more different multiplex gRNAs, cleaving a double strand of the proviral DNA at a first target protospacer sequence with the CRISPR-associated endonuclease, cleaving a double strand of the proviral DNA at a second target protospacer sequence with the CRISPR-associated endonuclease, excising an entire HIV-1 proviral genome, and eradicating the HIV-1 proviral DNA from the host cell.
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公开(公告)号：US20180236046A1
公开(公告)日：2018-08-23
申请号：US15885942
申请日：2018-02-01
Inventor： Kamel KHALILI , Wenhui HU
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 composition for use in inactivating a proviral DNA integrated into the genome of a host cell latently infected with a retrovirus including an isolated nucleic acid encoding a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, an isolated nucleic acid sequence encoding a first guide RNA (gRNA) having a first spacer sequence that is complementary to a first target protospacer sequence in a proviral DNA, and an isolated nucleic acid sequence encoding a second gRNA having a second spacer sequence that is complementary to a second target protospacer sequence in the proviral DNA, wherein said first target protospacer sequence and said second target protospacer sequence are situated in a long terminal repeat (LTR) of the proviral DNA. A pharmaceutical composition for use in inactivating a proviral DNA integrated into the genome of a host cell latently infected with a retrovirus.
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公开(公告)号：US20180228874A1
公开(公告)日：2018-08-16
申请号：US15904730
申请日：2018-02-26
Inventor： Kamel KHALILI , Wenhui HU
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 pharmaceutical composition for use in inactivating an HIV-1 proviral DNA integrated into the genome of a host cell latently infected with a retrovirus including a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, and two or more different multiplex 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) of the HIV-1 proviral DNA, whereby treating the host cell with the composition cleaves a double strand of the HIV-1 proviral DNA at a first target protospacer sequence with the CRISPR-associated endonuclease and cleaves a double strand of the HIV-1 proviral DNA at a second target protospacer sequence with the CRISPR-associated endonuclease and thereby excises an entire HIV-1 proviral genome and eradicates the HIV-1 proviral DNA from the host cell, and a pharmaceutically acceptable carrier.
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公开(公告)号：US20180207243A1
公开(公告)日：2018-07-26
申请号：US15921731
申请日：2018-03-15
Inventor： Kamel KHALILI , Wenhui HU
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 treating a subject having or at risk for having an HIV-1 virus infection, by administering to the subject a therapeutically effective amount of a composition comprising a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease, and two or more different multiplex 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) of proviral DNA of the virus that is unique from the genome of the host cell, cleaving a double strand of the proviral DNA at a first target protospacer sequence with the CRISPR-associated endonuclease, cleaving a double strand of the proviral DNA at a second target protospacer sequence with the CRISPR-associated endonuclease, excising an entire HIV-1 proviral genome, eradicating the HIV-1 proviral DNA from the host cell, and causing neither genotoxicity nor off-target editing to the host.
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