摘要:
Provided herein are recombinant poxviruses comprising heterologous or native nucleic acids specifying excess double-stranded RNA (dsRNA) early in infection, which may further comprise heterologous nucleic acids encoding one or more costimulatory molecules, and/or heterologous nucleic acids encoding one or more infectious disease-associated antigens or tumor-associated antigens, as well as pharmaceutical compositions comprising such recombinant poxviruses and methods and uses thereof. The recombinant poxviruses provided herein enhance innate and adaptive immune activation in subjects compared to identical recombinant poxviruses lacking heterologous or native transcription units specifying excess early dsRNA.
摘要:
The disclosure describes replication competent retroviral vectors (RCR) for gene therapy and gene delivery. The RCR includes an IRES sequence having 6-7 A's in A-bulge of the bifurcation region. The disclosure provides recombinant replication competent retrovirus comprising: a retroviral GAG protein; a retroviral POL protein; a retroviral envelope; a retroviral polynucleotide comprising Long-Terminal Repeat (LTR) sequences at the 3' end of the retroviral polynucleotide sequence, a promoter sequence at the 5' end of the retroviral polynucleotide.
摘要:
According to the present invention, an AAV vector having a higher titer compared with those of conventional ones can be produced using a cell into which a nucleic acid capable of expressing miRNA is introduced artificially. An AAV vector produced using the cell and a composition containing the viral vector as an active ingredient are very useful as gene transfer means in the studies or clinical practice of gene therapies.
摘要:
The present invention relates to compounds for their use in the prevention and/or treatment of a fibrotic disease in a subject, in particular to agents selected from the group consisting of miR-9, a variant thereof, an agent inducing the expression of miR-9 and an agent inducing the expression of a variant of miR-9, as well as to a diagnostic method of a fibrotic disease in a subject based on the miR-9 expression level in a sample from said subject.
摘要:
This invention is directed to an RNA interference (RNAi) agent and the use of that RNAi agent to treat Age-related Macular Degeneration, as well as pharmaceutical compositions containing the RNAi agents of the invention. The RNAi agent is a DNA-directed RNA interference (ddRNAi) agent (being an RNA molecule), together with an expression cassette or construct to express that agent in a cell (including in vivo), for inhibiting, preventing or reducing expression of an AMD associated gene. Preferably that AMD associated gene is one that is associated with wet AMD.
摘要:
The present invention is directed to RNA interference (RNAi) molecules targeted against a nucleic acid sequence, and methods of using these RNAi molecules to reduce off-target toxicity.