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公开(公告)号：US20180236044A1
公开(公告)日：2018-08-23
申请号：US15885936
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 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 proviral DNA harbored by a subject, designing two or more different guide RNAs (gRNAs) complementary to the proviral DNA sequences in the subject, treating the subject's host cells 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) of the proviral DNA, and inactivating the proviral DNA.
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公开(公告)号：US20180236042A1
公开(公告)日：2018-08-23
申请号：US15882207
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 at risk for having a virus infection, by administering to the subject a prophylactically 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 preventing a retroviral infection.
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公开(公告)号：US20230390357A1
公开(公告)日：2023-12-07
申请号：US18052863
申请日：2022-11-04
Inventor： Arthur M. FELDMAN , Joseph Y. CHEUNG , Kamel KHALILI
CPC classification number: A61K38/1709 , A61K31/69 , A61K45/06 , A61K38/05 , A61P9/10 , A61K9/0019 , C12N15/85
Abstract: The invention provides methods of treating ischemia/reperfusion injury in a subject. In one example, a method comprises administering a therapeutically effective amount of a pharmaceutical composition that increases levels of Bcl2-associated athanogene 3 (BAG3) polypeptide in ischemic tissue. The invention also provides methods of treating a subject at risk of ischemia/reperfusion injury. In one example, a method comprises administering a therapeutically effective amount of a pharmaceutical composition that increases levels of Bcl2-associated athanogene 3 (BAG3) polypeptide. In the invention methods, in one example, a pharmaceutical composition comprises a nucleic acid encoding BAG3 polypeptide.
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公开(公告)号：US20220380812A1
公开(公告)日：2022-12-01
申请号：US17776120
申请日：2020-11-11
Inventor： Kamel KHALILI , Hassen S. WOLLEBO , Jennifer GORDON , Ilker K. SARIYER
Abstract: Provided herein are gene editing compositions and methods that effectively modulate and/or edit a JCV genome. The effective modulation and/or editing is, in an aspect, achieved by gene editing compositions targeting a NCCR region, an early coding gene, and/or a late coding gene.
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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：US20180169194A1
公开(公告)日：2018-06-21
申请号：US15884427
申请日：2018-01-31
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 a retroviral 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 retrovirus, and eradicating the retroviral infection. A method of immunizing a subject at risk of retroviral infection, by administering a prophylactically effective amount of 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) of proviral DNA of a retrovirus to the subject, and preventing retroviral infection in the subject.
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18.发明公开公开(公告)号：US20240271128A1
公开(公告)日：2024-08-15
申请号：US18247523
申请日：2020-11-11
Inventor： Kamel KHALILI
CPC classification number: C12N15/111 , C12N9/22 , C12N15/86 , C12N2310/20 , C12N2750/14143
Abstract: The present disclosure relates to compositions and methods for the inhibition of the infectivity of a herpesvirus. The present disclosure relates in general to compositions and methods of treating or eradicating Herpes Simplex virus infections. The disclosure relates in particular to targeting of Herpes Simplex virus genes by gene editing complexes.
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公开(公告)号：US20190038770A1
公开(公告)日：2019-02-07
申请号：US16072636
申请日：2017-01-24
Applicant: EXCISION BIOTHERAPEUTICS,INC. , Temple University of the Commonwealth System of Higher Education
Inventor： Kamel KHALILI , Thomas MALCOLM
IPC: A61K48/00 , C12N15/66 , C12N15/113 , C12N15/861 , A61P31/20
Abstract: The present invention includes methods and compositions for elimination of polyoma viruses, such as John Cunningham Virus (JVC), from host cells, and the treatment of polyoma-virus related diseases, such as progressive multifocal leukoencephalopathy (PML). The compositions include 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 polyoma virus.
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公开(公告)号：US20190032057A1
公开(公告)日：2019-01-31
申请号：US16072589
申请日：2017-01-24
Applicant: EXCISION BIOTHERAPEUTICS, INC. , Temple University of the Commonwealth System of Higher Education
Inventor： Kamel KHALILI , Thomas Malcolm
IPC: C12N15/113 , C12N15/10 , C12Q1/70 , A61P31/18 , C12N15/86 , C12N9/22 , A61K31/711 , C12N15/70
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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