The invention relates to therapeutic compositions for disorders associated with haploinsufficiency. The invention provides antisense oligonucleotides useful for treating early-onset epileptic encephalopathy by promoting expression of Syntaxin-binding protein 1 (STXBP1). The invention provides compositions that include synthetic antisense oligonucleotides (ASOs) that prevent certain miRNAs from interfering with production of the STXBP1 protein or bind to the 5′-UTR of the STXBP1 transcript and augment translation of the STXBP1 protein. When the composition is delivered to a patient with STXBP1 haploinsufficiency, the ASOs prevent miRNA from downregulating synthesis of STXBP1 protein.

Molecular beacons and developmental methods related thereto. Methods include obtaining a nucleotide sequence for an aptamer that binds to a target analyte. The aptamer comprises a binding domain nucleotide sequence, a first domain nucleotide sequence, and a displacement domain nucleotide sequence complementary to the first domain nucleotide sequence. A molecular beacon is developed based on the nucleotide sequence of the aptamer by preserving the binding domain nucleotide sequence and truncating or extending one or both of the first domain nucleotide sequence or the displacement domain nucleotide sequence. The resultant molecular beacon is developed such that the molecular beacon comprises a Gibbs free energy value that is greater than the Gibbs free energy value of the aptamer.

The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

Disclosed are methods of using a microRNA-210 inhibitor in preparation of drugs for treating inflammatory skin diseases. The present inventor has demonstrated through a large number of experiments that in vitro inhibition of microRNA-210 expression can significantly enhance the expression of its target gene STAT6, thereby inhibiting proliferation and chemokine CCL20 secretion of keratinocytes, further inhibiting chemotactic T cell migration towards skin lesion, and also inhibiting differentiation of T.sub.H1 and T.sub.H17. MicroRNA-210 knockout and intradermal injection of a microRNA-210 inhibitor (cholesterol-modified antagomiR-210) on a skin lesion specifically inhibit the expression of microRNA-210, so that skin inflammation in mice can be significantly inhibited, and T cell immune imbalance is mitigated. The present invention provides a new pathophysiological mechanism for inflammatory skin diseases and provides a new strategy for preparing drugs for treating inflammatory skin diseases.

The present disclosure relates to a pharmaceutical composition for preventing or treating a brain disease, more particularly to a pharmaceutical composition for preventing or treating a brain disease, which contains a miR-485-3p inhibitor, and a method for screening an agent for preventing or treating a brain disease, which includes a step of measuring the expression level of miR-485-3p. Because the composition for treating a brain disease, which contains a miR-485-3p inhibitor, can restore the ELAVL2 protein unlike the exiting therapeutic agents for Alzheimer's disease, which are limited only to alleviating symptoms by inducing decreased expression of amyloid beta 42, the present disclosure can fundamentally treat various diseases caused by decreased expression of ELAVL2, such as Alzheimer's disease, autism spectrum disorder, mental retardation, amyotrophic lateral sclerosis, etc. Therefore, the present disclosure is useful for treating brain diseases including Alzheimer's disease fundamentally.

Disclosed are nucleic acid nanoparticles, a pharmaceutical composition comprising the same, a drug comprising doxorubicin and a preparation method thereof. The nucleic acid nanoparticles have a nucleic acid structural domain, the nucleic acid structural domain includes a sequence a, a sequence b and a sequence c; the sequence a includes a sequence a1 or a sequence obtained by insertion, deletion or substitution of at least one base in the sequence a1, the sequence b includes a sequence b1 or a sequence obtained by insertion, deletion or substitution of at least one base in the sequence b1 and the sequence c includes a sequence d or a sequence obtained by insertion, deletion or substitution of at least one base in the sequence.

The present invention concerns the use of antisense oligonucleotides to induce the death of several types of human cancer cells by silencing human L1-MET, which is a non coding transcript specifically transcribed in tumour cells.

This invention relates to polycomb-associated RNAs, libraries and fragments of those RNAs, inhibitory nucleic acids and methods and compositions for targeting RNAs, and methods of use thereof.

The present disclosure relates generally to modulation of genomic complexes via modulation of non-genomic components such as, non-coding RNAs.

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.