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Excision of retroviral nucleic acid sequences

A retrovirus and nucleic acid sequence technology, applied in the field of excision of retrovirus nucleic acid sequences, can solve problems such as inability to eliminate viruses

Pending Publication Date: 2019-03-01
TEMPLE UNIVERSITY
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

However, treatment fails to eliminate virus from latently infected cells (Kulpa DA, Chomont N.J VirusErad 2015;1:59-66)

Method used

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  • Excision of retroviral nucleic acid sequences
  • Excision of retroviral nucleic acid sequences
  • Excision of retroviral nucleic acid sequences

Examples

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preparation example Construction

[0203] The methods of the invention can be expressed in the manufacture of medicaments. Accordingly, the present invention includes the use of the agents and compositions described herein for the manufacture of a medicament. The compounds described herein are useful in therapeutic compositions and regimens or in the manufacture of medicaments for the treatment of the diseases or conditions described herein.

[0204] Any of the compositions described herein can be administered to any part of the host's body for subsequent delivery to target cells. Compositions can be delivered to the brain, cerebrospinal fluid, joints, nasal mucosa, blood, lungs, intestines, muscle tissue, skin, or peritoneal cavity of a mammal, but are not limited thereto. In terms of route of delivery, the compositions can be injected intravenously, intracranially, intraperitoneally, intramuscularly, subcutaneously, intramuscularly, rectally, intravaginally, intrathecally, intratracheally, intradermally or t...

Embodiment 1

[0231] Example 1: Excision of HIV-1 DNA by Gene Editing: In Vivo Studies

[0232] A CRISPR / Cas9 gene editing strategy was developed to eliminate integrated HIV-1 DNA sequences from latently infected human cells and animal disease models. The efficiency of HIV-1 DNA elimination was studied from mouse tissues carrying the viral genome.

[0233] Materials and methods

[0234] Construction of AAV9 delivery vector. The background plastid used to generate the AAV delivery system was px601-AAV-CMV::NLS-SaCas9-NLS-3xHA-bGHpA;U6::Bsa1-sgRNA, abbreviated as "pX601" (Ran FA et al., Nature 2015; 520 ):186-191). Oligonucleotides containing the DNA sequences corresponding to the LTR and Gag targets were cloned in front of the U6 promoter at the Bsa1 site in pX601. The gRNA sequences targeting LTR and Gag are shown in Figure 1A In, the PAM sequences are in bold as shown below: gRNA LTR-1: GGA TCA GAT ATC CAC TGA CCT TTG GAT; gRNA Gag D: GGA TAG ATG TAA AAGACA CCA AGG AGG. After confirm...

Embodiment 2

[0248] Example 2: In vivo eradication of HIV provirus by SaCas9 and polyploid sgRNA in a preclinical animal model.

[0249] In this study, combinations of sgRNAs targeting conserved HIV-1 regulatory and structural regions for efficient eradication of the HIV-1 genome using the saCas9 / sgRNA system were optimized and tested with HIV-diploid relative quadruple Feasibility and efficiency of sgRNA excision with saCas9 in HIV-1 Tg26 transgenic mice and EcoHIV enhanced firefly luciferase (eLuc)-infected mice in 1 primary animal (Rabinovich, B.A. et al. (2008) Proc Natl AcadSci USA 105, 14342-14346; Potash, M.J. et al. Proc Natl Acad Sci USA 102, 3760-3765, doi:10.1073 / pnas.0500649102 (2005)). Furthermore, it was demonstrated that polyploid sgRNA / saCas9 delivered by an all-in-one AAV vector could be used as pre-exposure prophylaxis (PrEP) in vivo.

[0250] Materials and methods

[0251] Bioinformatics design of highly efficient gRNAs. Efficient gRNA design was performed using the B...

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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.

Description

[0001] Statement Regarding Federally Funded Research [0002] This invention was made with partial government support under Grant Numbers P30MH092177 and R01NS087971 awarded by the National Institutes of Health. The US Government has certain rights in this invention. technical field [0003] Embodiments of the invention relate to gene editing complexes and in vivo delivery vehicles for preventing or treating retroviral infections. In particular, the gene editing complex comprises a clustered regularly interspaced short palindromic repeat (CRISPR)-related endonuclease encoded by a recombinant viral vector. Background technique [0004] HIV / AIDS remains a major public health problem with more than 40 million people infected worldwide and new infections consistently exceeding 2 million / year (Ade jumo OA et al, J Int AIDS Soc 2015;18:20049). Combination antiretroviral therapy (cART) effectively controls ongoing viral replication and can restore lost CD4 + number of T cells. ...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/11C12N15/113C12N9/22A61K48/00
CPCA61K48/00C12N9/22C12N15/1132C12N2310/20C07K2319/09A01K2227/105A01K2267/0337C12N2750/14143A61P31/14A61P31/18C12N15/11
Inventor K·哈利利W·胡
Owner TEMPLE UNIVERSITY
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