Abstract: Provided is an oligonucleotide modulator for preventing or treating dystrophin-deficient-related disorders (DDD) including Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). The oligonucleotide modulator comprises a sense nucleic acid strand and an antisense nucleic acid strand, wherein the sense nucleic acid strand and the antisense nucleic acid strand are independently an oligonucleotide strand of 16 to 35 nucleotides in length, in which one nucleotide strand has at least 75% base homology or complementarity to a target selected from a promoter region of a target gene UTRN.

Abstract: A double-stranded nucleic acid molecule, in particular a small activating nucleic acid molecule, and the use of the double-stranded nucleic acid molecule in the preparation of a drug, in particular in the preparation of a drug for activating a p21 gene and in the treatment or alleviation of proliferative vitreoretinopathy. The small activating nucleic acid molecule contains a first oligonucleotide chain and a second oligonucleotide chain which form a double-stranded structure by means of complete complementation or incomplete complementation, wherein the first oligonucleotide chain has at least 75% homology or complementarity with any continuous fragment with a length of 16-35 nucleotides of the promoter region of a human p21 gene.

Abstract: Provided herein are multi-valent oligonucleotide agents comprising two or more functional oligonucleotide units independently selected from single-stranded antisense oligonucleotides (ASOs, such as gapmers, mixmers and steric block ASOs) and duplex (double-stranded) RNAs (dsRNAs, such as siRNA and saRNA), and methods for the preparation thereof. Also provided herein are products, comprising the multi-valent oligonucleotide agents, and methods of using the multi-valent oligonucleotide agents or products in treatment of diseases (such as spinal muscular atrophy (SMA) and cancers).

Abstract: Provided is a small activating RNA used for promoting an expression level of a target gene. The small activating RNA is composed of a sense oligonucleotide chain and an antisense oligonucleotide chain. A nucleotide of the sense or antisense oligonucleotide chain has 2?-fluoro, 2?-methoxy, and a phosphorothioate modification to the nucleotide backbone.

Abstract: Provided herein are methods and compositions related to combinations of (a) agents that increase the expression of SMN2 gene or protein, and (b) modulators of SMN2 mRNA splicing or stability that increase the production of functional SMN2 mRNA and SMN protein, and their use in treating SMA and related conditions or diseases. In certain embodiments, the methods relate to using SMN2 saRNA and SMN2 mRNA modulators for diminishing the symptoms of SMA.

Abstract: Provided is a double-stranded nucleic acid molecule, in particular to a small activating nucleic acid molecule, wherein the small activating nucleic acid molecule may be a nucleic acid molecule targeting MITF gene and at least comprises a first oligonucleotide strand. Further provided are compositions or formulations comprising the small activating nucleic acid molecule and uses thereof. The small activating nucleic acid molecule, or the composition or formulation comprising the small activating nucleic acid molecule, can be used to activate or upregulate the MITF gene expression in a cell, and treat a disease or condition related to insufficient or decreased MITF protein expression.

Abstract: The present invention relates to a small activating nucleic acid molecules and uses thereof for treating diseases and conditions, such as thrombocytopenia, related to THPO protein deficiency or insufficiency. As described herein, small activating nucleic acid molecules can be double-stranded or single-stranded RNA molecules targeting the promoter region of the Thpo/THPO gene through an RNA activation mechanism and comprise a first nucleic acid strand and a second nucleic acid strand. The double-stranded RNA molecule targeting the promoter region of the Thpo/THPO gene comprises two nucleic acid strands of 16 to 35 nucleotides in length, wherein one nucleic acid strand has at least 75% homology or complementarity to a target selected from the promoter region of the Thpo/THPO gene.

Abstract: The present invention relates to oligomeric nucleic acid molecules and uses thereof for treating hearing loss. The present invention relates to small activating nucleic acid molecules for treating hearing loss. The small activating nucleic acid molecules of the present invention can be double-stranded or single-stranded RNA molecules targeting the promoter region of an ATOH1 gene comprising a first nucleic acid strand and a second nucleic acid strand. The double-stranded RNA molecule targeting the promoter region of the ATOH1 gene comprises two nucleic acid strands of 16 to 35 nucleotides in length, wherein one nucleic acid strand has at least 75% homology or complementarity to a target selected from the promoter region of the ATOH1 gene.

Abstract: The present invention relates to compositions of small activating nucleic acid molecules for increasing the expression of HMBS gene and a use thereof. The small activating nucleic acid molecule can be a double-stranded or single-stranded RNA molecule targeting the promoter region of the HMBS gene. The first nucleic acid strand and the second nucleic acid strand each contain a complementary region, and the complementary regions can form a double-stranded nucleic acid structure, which can promote the expression of the HMBS gene. The first nucleic acid strand or the second nucleic acid strand independently have a length of 16 to 35 nucleotides. The 3? terminus of the two oligonucleotide strands may have an overhang of 0 to 6 nucleotides in length.

Abstract: Provided are small activating nucleic acid molecules for skin care and uses thereof. The small activating nucleic acid molecule of the present invention comprises two oligonucleotide strands of 16 to 35 nucleotides in length, wherein one nucleotide strand has at least 75% homology or complementarity to a target selected from a promoter region of a target gene. Also provided are skin care products comprising a small activating nucleic acid molecule targeting the promoter region of the AQP3, AQP9, ELN, COL1A1, COL1A2, COL3A1, HAS1, HAS2, HAS3 or MFAP2 genes or a nucleic acid encoding the same and optionally an carrier or other effective ingredients. Further provided are methods for upregulating the expression of a target gene in cells using the small activating nucleic acid molecule or the nucleic acid encoding the same and improving skin conditions using the skin care products.

Abstract: The present invention relates to oligomeric nucleic acids and uses thereof for the treatment of tumors. The oligomeric nucleic acid for tumor treatment provided by the present application can be small activating nucleic acid molecules. A small activating nucleic acid molecule of the present invention can be a double-stranded or single-stranded RNA molecule targeting the promoter region of an LHPP gene comprising a first nucleic acid strand and a second nucleic acid strand. The double-stranded RNA molecule targeting the promoter region of the LHPP gene comprises two nucleic acid strands of 16 to 35 nucleotides in length, wherein one of the nucleic acid strands has at least 75% homology or complementarity to a target selected from the promoter region of the LHPP gene.

Abstract: The present invention relates to a small activating nucleic acid molecule for treating spinal muscular atrophy and use thereof. The small activating nucleic acid molecule comprises a sense nucleic acid strand and an antisense nucleic acid strand, wherein the sense nucleic acid strand and the antisense nucleic acid strand are independently an oligonucleotide strand of 16 to 35 nucleotides in length, in which one nucleotide strand has at least 75% base homology or complementarity to a target selected from a promoter region of a target gene SMN2.

Abstract: saRNAs are provided in the present invention. The saRNAs are composed of a first oligonucleotide strand containing 17 to 30 nucleotides and a second oligonucleotide strand containing 17 to 30 nucleotides. Sequences of at least 15 nucleotides in length are complementary in the two oligonucleotide strands, and the unpaired terminal nucleotides form overhangs. The first oligonucleotide strand or the second oligonucleotide strand has more than 75% homology or complementarity with any continuous fragment of 16 to 35 nucleotides in length in the promoter of the target gene. The second oligonucleotide strand has an overhang composed of 1 to 4 nucleotides at 3? end. saRNAs of the present invention can upregulate target gene expression more effectively while reducing off-target effects.

Abstract: The present invention provides a saRNA for activating or upregulating human p21 expression, the saRNA comprises a sense oligonucleotide strand and an antisense oligonucleotide strand, and the sense nucleic acid strand or the antisense nucleic acid strand has more than 75% homology with any continuous fragment of 16 to 35 nucleotides in length in the target gene sequence of a human p21 promoter, wherein the target nucleic acid sequence of the human p21 promoter is selected from a group consisting of SEQ ID NOs: 5, 6, 7, 8, 9, 10, 11, and 12.

Abstract: The present invention provides compositions, pharmaceutical preparations, kits and methods for increasing expression of a gene product in a cell by contacting the cell with a small activating RNA (saRNA) molecule comprising a ribonucleic strand that is complementary to a non-coding nucleic acid sequence of the gene.

Abstract: The present invention provides compositions, pharmaceutical preparations, kits and methods for increasing expression of a gene product in a cell by contacting the cell with a small activating RNA (saRNA) molecule comprising a ribonucleic strand that is complementary to a non-coding nucleic acid sequence of the gene.

Abstract: The present invention provides compositions, pharmaceutical preparations, kits and methods for increasing expression of a gene product in a cell by contacting the cell with a small activating RNA (saRNA) molecule comprising a ribonucleic strand that is complementary to a non-coding nucleic acid sequence of the gene.

Abstract: The present invention provides compositions, pharmaceutical preparations, kits and methods for increasing expression of a gene product in a cell by contacting the cell with a small activating RNA (saRNA) molecule comprising a ribonucleic strand that is complementary to a non-coding nucleic acid sequence of the gene.