RNAs, such as miRNA and siRNA, and their use in treating complement-mediated disorders, are described.

Provided is a nucleic acid construct for expressing a gene of interest, the nucleic acid construct comprising, in order from 5′-terminus, each sequence of: (a) a 5′ long terminal repeat (LTR) sequence derived from a retrovirus; (b) a packaging signal sequence (IV) derived from a retrovirus; (c) a sequence or multiple cloning site of the gene of interest; (d) a post-transcriptional regulatory sequence (PRE); (e) an siRNA generating sequence which forms at least one stem-loop structure and in which RNA, which induces RNA interference in mammalian cells, is transcribed; and (f) a 3′ LTR sequence derived form a retrovirus.

Provided are a precursor miRNA and application thereof in a tumor treatment. The precursor miRNA from the 5′ end to 3′ end has a structure presented as formula I:

##STR00001##

B1 is anti-miRNA-214-5p; B2 is an essentially complementary sequence or a totally complementary sequence to B1, and B2 and C are not complementary; C is a sequence having a stem-loop structure; A1 and A2 are respectively RNA sequences having no or 4-5 bases freely selected bases respectively; the precursor miRNA shown can be processed to form anti-miRNA-214 in a host, and only anti-miRNA-214-5p but not anti-miRNA-214-3p is expressed in the anti-miRNA-214.

Compositions, non-viral vectors, recombinant viruses, and recombinant viral vectors for inhibiting ΔFosB expression or activity in a cell and for treating dyskinesia in a subject (e.g., a human patient having Parkinson's disease and Levodopa-induced dyskinesia) include a nucleic acid sequence encoding a shRNA specific for ΔFosB. Methods of using these compositions, non-viral vectors, recombinant viruses, and recombinant viral vectors are also described herein. These compositions, non-viral vectors, recombinant viruses, and recombinant viral vectors and methods of use provide novel therapies for dyskinesia based on the reduction of ΔFosB expression and/or activity.

Among the various aspects of the present disclosure is the provision of methods for treating pathologic calcification or bone formation and methods of inhibiting KIF26B that include administering a therapeutically effective amount of a synthetic nucleic acid against KIF26B. Compositions comprising a small hairpin RNA (shRNA) against KIF26B are also provided.

A lentiviral vector system for expressing a lentiviral particle is disclosed. The lentiviral vector system includes a therapeutic vector, an envelope plasmid, and at least one helper plasmid. The lentiviral vector system can produce a lentiviral particle for inhibiting PARP expression in neuron cells of a subject afflicted with Parkinson's disease.

Disclosed herein are peptide sequences capable of directing adeno-associated viruses (AAV) to target specific environments, for example the nervous system and the heart, in a subject. Also disclosed are AAVs having non-naturally occurring capsid proteins comprising the disclosed peptide sequences, and methods of using the AAVs to treat diseases.

The present invention provides a siRNA for inhibiting HTT gene expression and a precursor thereof, an expression vector comprising the siRNA or the precursor thereof, application thereof in preparation of a drug for treating Huntington's disease, and a pharmaceutical formulation and pharmaceutical composition containing the siRNA, the precursor or the expression vector. The siRNA is selected from the following: SEQ ID NO:1, SEQ ID NO:2. SEQ ID NO:3, SEQ ID NO:4, SEQ ID NO:5, or a combination thereof.

This disclosure relates to RNA interference (RNAi) reagents for treatment of hepatitis B virus (HBV) infection, compositions comprising same, and use thereof to treat individuals infected with HBV.

The present invention relates generally to immunization and immunotherapy for the treatment or prevention of HIV. In particular, the methods include in vivo and/or ex vivo enrichment of HIV-specific CD4+ T cells.