Abstract: The present disclosure relates to an RNAi-inducing nucleic acid molecule and use thereof. An aspect of the disclosure relates to an RNAi-inducing nucleic acid molecule for inhibiting expression of myeloid differentiation primary response gene 88 (MyD88). Another aspect of the present disclosure relates to a pharmaceutical composition for treating or preventing age-related macular degeneration, comprising the RNAi-inducing nucleic acid molecule.

Abstract: Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.

Abstract: The present invention is directed to provide novel RNA molecules, chimeric NA molecules, double-stranded RNA molecules, and double-stranded chimeric NA molecules. Specifically, an embodiment of the present invention is an RNA molecule for RNA interference to target a mutant allele with a point mutation, in which (1) the molecule has a nucleotide sequence complementary to a nucleotide sequence of a coding region of the mutant allele; and (2) when counted from the base at the 5?-end in a nucleotide sequence complementary to a nucleotide sequence of the mutant allele, (2-1) a base at position 5 or 6 is mismatched to a base in the mutant allele; (2-2) a position 10 or 11 corresponds to the position of the point mutation; and (2-3) a group at the 2?-position of a pentose at positions 6-8 or positions 7 and 8 is modified with, e.g., OCH3. In this RNA molecule, one or more ribonucleotides may be replaced by, e.g., a deoxyribonucleotide. The molecule may form a double-stranded RNA with a complementary strand.

Abstract: The present invention relates to RNAi constructs for reducing expression of the PNPLA3 gene. Methods of using such RNAi constructs to treat or prevent liver disease, nonalcoholic fatty liver disease (NAFLD) are also described.

Abstract: Compositions and methods for editing, e.g., introducing double-stranded breaks, within the KLKB1 gene are provided. Compositions and methods for treating subjects having hereditary angioedema (HAE), are provided.

Abstract: Provided are compositions comprising an oligonucleotide that targets Angiopoietin-like 4 (ANGPTL4). The oligonucleotide may include a small interfering RNA (siRNA) or an antisense oligonucleotide (ASO). Also provided herein are methods of treating a metabolic or cardiovascular disorder by providing an oligonucleotide that targets ANGPTL4 to a subject in need thereof.

Abstract: The present invention relates to aptamers that specifically bind to cancer stem cells. The aptamers according to the present invention specifically bind to cancer stem cells and reduce cell adhesion ability, cell proliferation, drug resistance and cell migration, which are characteristics of cancer stem cells, thus having excellent anticancer effects. Therefore, the aptamer may be used in various ways in the fields of cancer diagnosis, prognosis prediction, and treatment.

Abstract: The present invention relates to an inhibitor of miR-129, relative compounds and pharmaceutical compositions for use in the treatment and/or prevention of amyotrophic lateral sclerosis and Alzheimer's disease. The invention also relates to a method for the diagnosis and/or prognosis of Alzheimer's disease in a subject or to identify a subject at risk to develop amyotrophic lateral sclerosis or Alzheimer's disease and to a method for the measuring the efficacy of a therapy for amyotrophic lateral sclerosis or for Alzheimer's disease and relative kits.

Abstract: The present disclosure is related to compositions and methods useful in treating heart conditions. The disclosed compositions and methods are based on gene therapies comprising a recombinant AAV vector for delivering two or more transgenes into the heart of a subject, wherein the transgenes encode an S100A1 protein and a cardiac Apoptosis Repressor with caspase recruitment Domain (cARC) apoptotic inhibitor, respectively. In various embodiments, the compositions and methods disclosed herein comprise vectors comprising S100A1 and/or cARC cDNA sequences that are codon-optimized for expression in humans. In various embodiments, the compositions and methods disclosed herein comprise vectors with improved packaging efficiencies. In some aspects, targeting multiple sources of one or more heart conditions can provide synergistic benefits during treatment.

Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of AGT RNA in a cell or subject, and in certain instances reducing the amount of AGT in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a cardiovascular disease. Such compound and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a RAAS pathway-related disease or disorder. Such symptoms and hallmarks include hypertension, chronic kidney disease, stroke, myocardial infarction, heart failure, valvular heart disease, aneurysms of the blood vessels, peripheral artery disease, and organ damage. Such cardiovascular diseases include hypertension, resistant hypertension, Marfan syndrome, and heart failure.

Abstract: The disclosure provides, inter alia, hybridized nucleic acid sequences and compounds comprising Toll-like receptor 9-binding nucleic acid sequences and nucleic acid sequences comprising a microRNA passenger strand sequence hybridized to a microRNA guide strand sequence; pharmaceutical compositions comprising the hybridized nucleic acid sequences and compounds; and the use of the hybridized nucleic acid sequences, compounds, and pharmaceutical compositions to treat medical conditions, such as cancer and inflammatory diseases.

Abstract: The present disclosure provides an anti-VEGF aptamer, an agent or composition comprising the anti-VEGF aptamer, as well as uses thereof.

Abstract: The invention relates to a nucleic acid aptamer with the capability of binding specifically to a and inhibiting TLR-4, to a complex comprising said aptamer and a functional group, as well as to pharmaceutical compositions thereof. The invention also relates to uses and methods for detecting TLR-4 and to uses and methods for inhibiting TLR-4. Finally, the invention also relates to an aptamer for use in manufacturing a drug for the treatment of a pathology characterized by an increase in expression of TLR4 and/or an increase in activation of TLR-4.

Abstract: The present invention relates to the discovery that the expression levels of some RNA molecules, comprising messenger RNA (mRNA), non-coding RNA (ncRNA) and/or microRNA (miRNA), and protein can be used as a diagnostic signature to predict or monitor the bone healing ability in an acutely injured subject or in a chronic nonunion subject. In certain embodiments, the invention relates to methods and compositions useful for differentiating between a nonunion, slow healing, and/or normal healing of a fractured bone and treatment recommendations. The invention further includes a kit comprising biomarker probes for assessing the bone healing ability in an acutely injured subject or in a nonunion subject after receiving therapeutic treatment.

Abstract: RNA editing is achieved using oligonucleotide constructs comprising (i) a targeting portion specific for a target nucleic acid sequence to be edited and (ii) a recruiting portion capable of binding and recruiting a nucleic acid editing entity naturally present in the cell. The nucleic acid editing entity, such as ADAR, is redirected to a preselected target site by means of the targeting portion, thereby promoting editing of preselected nucleotide residues in a region of the target RNA which corresponds to the targeting portion.

Abstract: The present invention discloses a SmartBac baculovirus expression system and application thereof. The system can comprise a acceptor plasmid (containing fragment A or fragments B and C) and a donor plasmid (containing fragment D); the fragment A contains a promoter, a sequence encoding a protease, a protease cleavage site, an insertion region of a gene encoding a target object to be expressed and a termination sequence; the fragment B contains a promoter, a sequence encoding a protease and a termination sequence; the fragment C contains a promoter, an insertion region of a gene encoding a target object to be expressed and a termination sequence; the fragment D contains a promoter, an insertion region of a gene encoding a target object to be expressed and a termination sequence.

Abstract: Described herein and RNA aptamers that specifically bind to tumor-infiltrating myeloid cells and uses thereof.

Abstract: The present invention relates to a composition for inhibiting the growth of cancer stem cells, and a use thereof. A WDR34 inhibitor of the present invention inhibits the conversion of cancer cells into cancer stem cells and exhibits activity of inhibiting self-renewal, invasion, and migration of cancer stem cells, and thus can be effectively used as a cancer cell growth or metastasis inhibitor or a cancer stem cell growth inhibitor.

Abstract: A method for in utero genome editing, the method comprising administering to a subject an adenoviral vector, wherein the subject is a fetus, the adenoviral vector comprising CRISPR-mediated base editor and a guide RNA (gRNA), the gRNA targeting a mutation in a therapeutic gene; and introducing a modified codon in the therapeutic gene by base editing the therapeutic gene, wherein the base editing is performed by the adenoviral vector, an adeno-associated viral vector or lipid based nanoparticle.

Abstract: Compositions and methods for editing, e.g., altering a DNA sequence, within the TRBC1, TRBC2 and/or TRAC genes are provided. Compositions and methods for immunotherapy are provided, for example.