公开(公告)号：US20210052709A1

公开(公告)日：2021-02-25

申请号：US17017037

申请日：2020-09-10

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

Inventor： Kamel KHALILI ,  Wenhui HU ,  Yonggang ZHANG

IPC: A61K38/46 ,  C12N15/113 ,  A61K48/00 ,  A61P31/18 ,  C12N9/22 ,  C12N15/11 ,  C12N15/90

Abstract: Compositions for specifically cleaving target sequences in retroviruses include nucleic acids encoding a Clustered Regularly Interspace Short Palindromic Repeat (CRISPR) associated endonuclease and a guide RNA sequence complementary to one or more target nucleic acid sequences in a retrovirus genome.

公开(公告)号：US20180303915A1

公开(公告)日：2018-10-25

申请号：US15918107

申请日：2018-03-12

Applicant: Temple University of the Commonwealth System of Higher Education

Inventor： Kamel Khalili ,  Wenhui HU

IPC: A61K38/46 ,  C12N15/11 ,  A61K35/12 ,  A61K48/00 ,  C12N7/00 ,  C12N9/22 ,  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 treating a subject at risk for having an HIV-1 virus infection, by administering to the subject a prophylactically effective amount of a composition comprising a 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, and eradicating the HIV-1 proviral DNA from the host cell and preventing HIV-1 retroviral infection.

公开(公告)号：US20180236045A1

公开(公告)日：2018-08-23

申请号：US15885940

申请日：2018-02-01

Applicant: Temple University of the Commonwealth System of Higher Education

Inventor： Kamel KHALILI ,  Wenhui HU

IPC: A61K38/46 ,  C12N7/00 ,  A61K9/00 ,  A61K35/12 ,  A61K45/06 ,  A61K48/00 ,  C12N9/22 ,  C12N15/11

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 preventing transmission of a retrovirus from a mother to her offspring, by treating the mother'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 preventing transmission of the proviral DNA to the offspring.

公开(公告)号：US20180236042A1

公开(公告)日：2018-08-23

申请号：US15882207

申请日：2018-01-29

Applicant: Temple University of the Commonwealth System of Higher Education

Inventor： Kamel KHALILI ,  Wenhui HU

IPC: A61K38/46 ,  C12N7/00 ,  A61K9/00 ,  A61K35/12 ,  A61K45/06 ,  A61K48/00 ,  C12N9/22 ,  C12N15/11

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.