摘要:
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
摘要:
The present invention relates to methods for the identification of anti-HIV miRNAs and anti-HIV pharmaceutical compounds using high-throughput screening methods, comprising: transfecting reporter cells with a panel of miRNAs, infecting the reporter cells with HIV, screening the cells to identify miRNAs that modulate HIV infection and identifying the specific pathways, nucleic acids and/or polypeptides that are targeted by the miRNAs. The invention further provides for the identification and screening of anti-HIV pharmaceutical compounds having known activity against the specific pathways, nucleic acids and/or polypeptides that are targeted by the miRNAs for efficacy in the treatment of HIV. The invention also provides for the use of miRNA mimics, miRNA inhibitors and pharmaceutical compounds (including oncology drugs and kinase inhibitors) in the treatment and/or prevention of HIV infection.
摘要:
Auto-recognizing therapeutic R/DNA chimeric nanoparticles (R/DNA NP) are described that are pairs of DNA/RNA hybrids where the DNA molecules have complementary toehold sequences that promote the re-association of the R/DNA NPs when mixed resulting in the formation of RNA/RNA duplexes that act as siRNAs.
摘要:
The present invention describes a method to inhibit replication of the human immunodeficiency virus (HIV) by negatively modulating or altering the cytoskeleton, more precisely the proteins forming the intermediate cytoskeletal filaments, wherein the said proteins are vimentin and/or keratin-10. The replication of the virus is inhibited in human cells by intervening in the structure of these proteins. The present invention is also related to the use of agents, which comprise peptides and/or interfering RNA and/or lipidic compounds, said agents producing a negative modulation or alteration of the cytoskeleton to prevent or to treat the HIV infection. The invention provides means and methods for altering the cytoskeleton/filament structure of cells, as a result of which the infection of human cells by HIV is disturbed and can even be completely inhibited. The cytoskeleton is altered by reducing the amount of vimentin and/or keratin (e.g. by transcriptional control using interfering RNA) or by using peptides that disrupt the cytoskeleton.
摘要:
The invention provides a set of nucleotides against HIV infection and their application for treatment and prevention of AIDS. The sequences of the nucleotides were shown in the sequences table. The invention was superior to the similar technology as following: High conserved RNA sequences were obtained by compare all the published HIV genome sequences. Double strand RNA sequences derived from the sequences can effectively inhibit the expression of target HIV gene. HIV gene expression can also be inhibited by double strand hairpin-like RNA transcribed in the cells harboring recombinant plasmid which contain the DNA sequences encoding the RNA sequences. Further, HIV gene expression can be knocked down by in cell transcribed double strand RNA corresponding to the RNA sequences encoded by recombinant adenovirus associated virus.
摘要:
The present invention relates to interfering RNA, in particular miRNAs capable of specifically blocking the replication of an HIV virus strain resistant to an antiretroviral compound, and to the use thereof for treating infections by said type of virus.
摘要:
The present invention concerns methods and reagents useful in modulating hepatitis C virus (HCV) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against hepatitis C virus (HCV) gene expression and/or activity. The small nucleic acid molecules are useful in the treatment and diagnosis of HCV infection, liver failure, hepatocellular carcinoma, cirrhosis and any other disease or condition that responds to modulation of HCV expression or activity.
摘要:
La présente invention concerne de nouveaux vecteurs, les compositions pharmaceutiques les contenant, et leurs utilisations thérapeutiques. Plus particulièrement, elle concerne de nouvelles molécules capables d'agir, de manière très sélective et très efficace, sur l'expression de gènes.