Provided herein are methods, compounds, and compositions useful for targeted delivery of compounds to non-native cells ectopically expressing cell surface receptors. Such methods, compounds, and compositions are useful, for example, in gene therapy mediated ectopic expression of cell surface receptors and targeted delivery of compounds, such as conjugated oligonucleotides, to the non-native cells ectopically expressing cell surface receptors. Such methods, compounds, and compositions can be useful, for example, to treat, prevent, delay or ameliorate disease in an individual by targeted reduction of a gene of interest in the non-native cell ectopically expressing cell surface receptors.

Provided is a polynucleotide in which an N-terminal region of TIF1y gene is codon-optimized, a recombinant vector including the polynucleotide, and a use thereof.

The present invention relates to methods and compositions for inhibiting metastatic spread of cancer and/or inhibiting progression of pre-existing metastatic disease in a subject using L1CAM inhibition.

Compositions containing MiniVectors and gene therapy uses, including long term repeated gene therapy uses, to treat liver fibrosis or liver cancer.

Provided herein are methods for improving memory or cognitive function in a subject by administering a composition to the brain of the subject, where the composition comprises: i) a compound that increases expression of MALAT-1 long non-coding RNA, ii) a MALAT-1 long-coding RNA nucleic acid sequence, or iii) at least one MALAT-1 derived piRNA nucleic acid sequence. Also provided herein are methods of screening candidate compounds for their ability to modulate the expression of MALAT-1 long non-coding RNA in brain cells. In certain embodiments, such identified modulators that increase expression are further administered to the brain of a lab animal to determine the impact of such modulators on learning and memory.

Methods for treating, and for identifying novel treatments for, neurodegenerative diseases, as well as animal and cellular models. The present disclosure shows that age dependent accumulation of genomic lesions leads to the production of RNA molecules within neurons that mimic viruses and intrinsically activate innate immune signaling, which triggers neurodegeneration. This hypothesis is supported by the results shown herein elucidating the mechanism of neurodegeneration in two mouse lines that specifically express different isoforms of the human amyloid precursor protein (hAPP) gene, which is associated with Alzheimer's disease (AD), exclusively in olfactory sensory neurons (OSNs) in the nose.

The present invention relates to a pharmaceutical composition for preventing or treating peripheral vascular disease, the composition comprising, as an active ingredient: (a) hepatocyte growth factor (HGF) or an isoform thereof, and stromal cell derived factor 1α (SDF-1α); or (b) a polynucleotide encoding the HGF and a polynucleotide encoding the SDF-1α. The peripheral vascular disease (for example, ischemic limb disease) can be more effectively prevented or treated through the significant promotion of vascular endothelial cell migration and angiogenesis in the case of singly using the composition of the present invention than in the case of using HGF, an isoform thereof, SDF-1α or a polynucleotide codes a protein thereof.

Disclosed are nanoparticles that are introduced into cells and express a specific protein and a manufacturing method thereof. More particularly, the present invention relates to mRNA nanoparticles, which increase the expression of a specific protein capable of stimulating the cellular immune system to induce cellular immune responses and are thus applicable to treat a variety of diseases, do not require passage across the nuclear envelope because a desired gene is delivered not as plasmid DNA itself but in the form of mRNA, thus improving the efficiency of protein expression, and the nanoparticles are generated through a one-step process with a relatively small amount of plasmid DNA via rolling circle transcription (RCT), thereby providing a simple and economical process for gene delivery. The present invention is also concerned with such mRNA nanoparticles.

A pharmaceutical composition includes an NAG-1 inhibitor as an active ingredient for inhibiting resistance against an anticancer drug of an ovarian cancer patient and a method of diagnosing prognosis of resistance against an anticancer drug of an ovarian cancer patient by using the NAG-1 inhibitor. It is found by controlling NAG-1 protein that NAG-1, which is overexpressed in an ovarian cancer patient and in an ovarian cancer stem cell having resistance against an anticancer drug, plays a key role in a chronic inflammatory reaction and resistance against an anticancer drug, and in this regard, NAG-1 can be used as a target gene for effective tumor therapy.

Methods for inducing skipping of exons, including exon 51 of the dystrophin gene. Oligonucleotides are used for inducing exon skipping and for treating Duchenne Muscular Dystrophy. Disclosed structures include: (1) h51AON1 (SEQ ID NO: 27; UCAA GGAA GAUG GCAU UUCU), which is 20 bases long, (2) h51AON2 (SEQ ID NO: 28; CCUC UGUG AUUU UAUA ACUU GAU), which is 23 bases long, and (3) the combination of h51AON2 and h45AON5 linked by 10 uracils (i.e., SEQ ID NO: 28 (CCUC UGUG AUUU UAUA ACUU GAU) linked to SEQ ID NO: 16 (GCCC AAUG CCAU CCUG G) by UUUU UUUU UU), which combination is 50 bases long.