Methods are provided for the prognosis, diagnosis and treatment of various pathological states, including cancer, chemotherapy resistance and dementia associated with Alzheimer's disease. The methods provided herein are based on the discovery that various proteins with a high level of sialylation are shown herein to be associated with disease states, such as, cancer, chemotherapy resistance and dementia associated with Alzheimer's disease. Such methods provide a lysosomal exocytosis activity profile comprising one or more values representing lysosomal exocytosis activity. Also provided herein, is the discovery that low lysosomal sialidase activity is associated with various pathological states. Thus, the methods also provide a lysosomal sialidase activity profile, comprising one or more values representing lysosomal sialidase activity. A lysosomal sialidase activity profile is one example of a lysosomal exocytosis activity profile.

The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.

Provided herein are methods, compounds, and compositions for reducing expression of an ANGPTL3 mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for reducing lipids and/or glucose in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one or more of cardiovascular disease and/or metabolic disease, or a symptom thereof, in an individual in need thereof.

The present invention relates to oligonucleotides that upregulate or restore the expression of progranulin in cells by targeting the promoter region of the progranulin gene. The invention further relates to pharmaceutical compositions and methods for the treatment of diseases associated with progranulin, specifically progranulin haploinsufficiency and neurological disorders.

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.

The present disclosure belongs to the technical field of biomedicine, and provides an engineered circular RNA circmiR-29b and use thereof in preparation of a medicine for treating muscle atrophy. The present disclosure provides a circular RNA circmiR-29b including an effective sequence and a random sequence, wherein 6-13 repetitions of the effective sequence are connected in series, the random sequence is inserted between the effective sequences, and the nucleotide sequence of the effective sequence is shown in SEQ ID NO: 1. The present disclosure delivers circmiR-29b to skeletal muscle by AAV8, enabling stable expression of circmiR-29b in the skeletal muscle, thereby effectively inhibiting various types of muscle atrophy. Therefore, the present disclosure also provides a gene therapy based on the AAV8 virus vector delivering the engineered circular RNA circmiR-29b to achieve the objective of treating muscle atrophy.

This invention relates to nanofibers comprising aptamers, which have increased stability and activity relative to free aptamers. The invention further relates to kill-switch nanofibers which disrupt the aptamer nanofibers. The invention further relates to methods of using the aptamer nanofibers and the kill-switch nanofibers to regulate the activity of extracellular targets recognized by the aptamers.

Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C. Additionally disclosed are antisense compounds and methods for treating, preventing, or ameliorating hypercholesterolemia and/or atherosclerosis. Further disclosed are antisense compounds and methods for decreasing coronary heart disease risk. Such methods include administering to an individual in need of treatment an antisense compound targeted to a PCSK9 nucleic acid. The antisense compounds administered include gapmer antisense oligonucleotides.

The present embodiments provide methods, compounds, and compositions useful for inhibiting APOL1 expression, which may be useful for treating, preventing, or ameliorating a disease associated with APOL1.

The present invention provides a method for assessing the possibility of onset or progression of chronic kidney allograft rejection or chronic kidney disease in a test subject, by using, as an indicator, the level of SYT17 protein in a urine-derived specimen collected from the test subject. The present invention also provides: a pharmaceutical composition for preventing and/or treating chronic kidney allograft rejection or chronic kidney disease, the pharmaceutical composition including at least one substance selected from the group consisting of a specific antibody to SYT17, a derivative of the specific antibody, and an inhibitory nucleic acid against SYT17; and a test kit for use in assessing the possibility of onset or progression of chronic kidney allograft rejection or chronic kidney disease, the test kit including a specific antibody to SYT17 or a derivative of the specific antibody.