24 results about "Mirna target" patented technology

The present application relates to an isolated nucleic acid comprising (i) an open reading frame encoding a protein that is not to be expressed in a specific cell but to be expressed in a cell differing from the specific cell and (ii) a miRNA target site, wherein the miRNA target site is recognized by a miRNA that is expressed in the specific cell but substantially not expressed in the cell differing from the specific cell. The present application further relates to a particle comprising the afore-mentioned isolated nucleic acid as well as a pharmaceutical composition comprising the afore-mentioned particle. The present application is also concerned with a method of expressing a protein in a cell substantially not expressing a miRNA but not expressing the protein in a cell expressing said miRNA, either in vitro or in vivo in a subject, the use of the afore-mentioned nucleic acid, particle and pharmaceutical composition to express a protein in a cell-specific manner; with a method of treating a B cell cancer, an immune cell and a kit related to the afore-mentioned aspects.

The invention belongs to the technical field of biological medicine, and relates to application of miRNA targeted regulation in treatment of acute respiratory distress syndrome, in particular to application of a miR-144 inhibitor in preparation of a medicine for improving glucocorticoid treatment sensitivity of an ARDS patient. Research on a lipopolysaccharide-induced ARDS rat model finds that miR-144 participates in regulation and control of glucocorticoid receptor beta (GR beta) and NF-kappa B signal pathways in the ARDS inflammatory reaction process, the number of neutrophils in alveolar lavage fluid and the level of inflammatory factors such as IL-6 and TNF-alpha can be remarkably reduced by using the miR-144 inhibitor and glucocorticoid for treatment, and the application of the miR-144 inhibitor to treatment of the pulmonary alveolar lavage fluid in the treatment of the pulmonary alveolar lavage fluid is broad. The wet-to-dry ratio and pathological injury score of lung tissues are reduced, and GR beta and NF-kappa B expression is inhibited, so that the anti-inflammatory effect of glucocorticoid is remarkably enhanced. The invention provides a treatment strategy for improving glucocorticoid resistance of ARDS patients by taking miR-144 as a target for the first time, and provides a new drug target and a technical scheme for accurate treatment of ARDS.

Provided herein are compositions and methods for treatment of Amyotrophic Lateral Sclerosis as well as Spinocerebellar Ataxia Type 2 by way of reducing levels of the ATXN2 gene expression. More specifically. miRNA compositions that target ATXN2 are able to reduce ATXN2 levels.

The invention concerns synthetic miRNAs targeting the TOX gene and uses thereof. Pharmaceutical compositions comprising such miRNAs and uses thereof in the medical field, in particular in the treatment of cancer diseases and immunotherapy, are also described.

The present application relates to the technical field of spleen delivery, and specifically discloses a spleen precise expression composition, a vaccine composition and application thereof. The spleen precise expression composition comprises: component (1): at least one nucleic acid comprising a nucleotide sequence encoding a target protein and one or more miRNA targeting binding sequences; component (2): a lipid nanoparticle composition which is easy to accumulate in the spleen. The spleen precise expression composition can be used for preparing a lipid nanoparticle or a drug or vaccine composition, and can be efficiently delivered into the spleen and its tissues and cells, and significantly reduces the expression of target genes in non-spleen organs, avoiding side effects such as inflammatory reactions or other off-target effects caused by undesired expression.

Provided are: a novel HMGB1 expression regulator including small extracellular vesicles which include a miRNA targeting a gene involved in HMGB1 expression and can regulate HMGB1 expression using the miRNA derived from the small extracellular vesicles; a prophylactic agent or therapeutic agent for acute lung injury, acute respiratory distress syndrome, or sepsis; and a method of ameliorating acute lung injury, acute respiratory distress syndrome, or sepsis. The small extracellular vesicle is preferably exosomes.

The present disclosure provides miRNA mimics targeting CDK4 and / or CDK6, and compositions comprising the same. Methods for the treatment of tumors, including but not limited to colon cancer, comprising administering a modified oligonucleotide comprising a miRNA are also disclosed herein.

A polynucleotide comprising at least one miR-124 target sequence, and / or at least one miR-338-3p target sequence, and / or at least one miR-31 target sequence, wherein the miRNA target sequences are operably linked to a transgene.

The invention concerns synthetic miRNAs targeting the CISH gene and uses thereof. Pharmaceutical compositions comprising such miRNAs and uses thereof in the medical field, in particular in the treatment of cancer diseases and in immunotherapy, are also described.

An isolated nucleic acid sequence including at least two copies of a miRNA target site of the miR183 family having a sequence as set forth in SEQ ID NO: 1, SEQ ID NO: 21, or SEQ ID NO: 24, or a sequence having at least 90% identity with any one of SEQ ID NO: 1, SEQ ID NO: 21, or SEQ ID NO: 24. Also a vector including a regulatory element having at least one copy of this miRNA target site of the miR183 family. The isolated nucleic acid sequence and the vector may be particularly useful for controlling the expression of a gene of interest, for example when designing and developing gene therapies.

The present disclosure provides miRNA mimics targeting CDK4 and / or CDK6, and compositions comprising the same. Methods for the treatment of tumors, including but not limited to colon cancer, comprising administering a modified oligonucleotide comprising a miRNA are also disclosed herein.

Microparticles that contain miRNA that controls the production of nitric oxide and are an agent for promoting the production of nitric oxide are new microparticles that enable controlling the expression of a protein and / or a gene related to erectile dysfunction; a preventive drug or a therapeutic drug for erectile dysfunction; a method for improving erectile dysfunction; it is preferable that the microparticles be exosomes; and it is preferable that the microparticles of the present disclosure contain miRNA targeting a gene concerning the expression of nitric oxide synthase as the miRNA that controls the production of nitric oxide, and be an agent for promoting the expression of nitric oxide synthase.

The invention discloses miR-285-3p for preventing and treating spodoptera frugiperda and application of the miR-285-3p. MiRNA, miR-285-3p targeting a key gene for growth and development of the spodoptera frugiperda is screened for the first time, and after an analogue agomiR-285-3p of the miRNA, miR-285-3p is injected, the agomiR-285-3p is found to be capable of inhibiting normal expression of ecdysone induced protein 74EF (Ecdyson-induced protein 74EF, Eip74EF) gene of the spodoptera frugiperda, and the agomiR-285-3p can be used for inhibiting the growth and development of the spodoptera frugiperda. Therefore, the growth and development of the spodoptera frugiperda larvae are slowed down, the feed intake is reduced, and the death rate is increased. In addition, by adding an analogue agomiR-285-3p into the feed, the growth of the spodoptera frugiperda can be inhibited, the food intake is reduced, and the death rate is increased. The invention provides a new target and technical support for establishing a new strategy for preventing and controlling the spodoptera frugiperda based on the miRNA.

The invention provides a novel miRNA target for treating AIDS (acquired immune deficiency syndrome) infection and cervical cancer and application of the novel miRNA target. The miRNA comprises miR-H1 (micro Ribonucleic Acid), miR-TAR (micro Ribonucleic Acid) and miR-N367, and is called HIV1-miRNAs for short. The HIV1-miRNAs exist in a cervical cancer patient suffering from HIV (Human Immunodeficiency Virus) infection. The HIV1-miRNAs can participate in the occurrence and development process of HIV combined cervical cancer by regulating and controlling tumor key pathways such as cell proliferation and inflammation in cervical cancer cells. The invention also provides regulatory molecules (including antisense nucleotides) for targeted regulation. The invention provides a new diagnosis and treatment way for clinical treatment and detection of HIV combined cervical cancer.

The application discloses a marker combination for diagnosing Alzheimer's disease and application and kit thereof.The marker of the application comprises STMN1 protein, STN1 mRNA and hsa- miR-9-5p in peripheral blood, STN1 the content of the mRNA and the miRNA targeting STN1 target genes hsa-miR-9- 5p in a peripheral blood sample is detected, data is input into a diagnosis model trained by Logistic regression fitting in advance, and thus, accurate screening and diagnosis of Alzheimer's disease can be realized.Compared with an invasive detection scheme such as cerebrospinal fluid, the application adopts peripheral blood sampling, is simple to operate, has lower detection cost and is suitable for clinical large-scale noninvasive screening.

The invention discloses miR-2c-5p for preventing and treating spodoptera frugiperda and application of miR-2c-5p. MiRNA and miR-2c-5p targeting a key gene for growth and development of the spodoptera frugiperda are screened for the first time, and after an analogue agomiR-2c-5p of the miRNA and miR-2c-5p is injected, the agomiR-2c-5p is found to be capable of inhibiting normal expression of ecdysone-induced protein 78C (Ecdyson-induced protein 78C, Eip78C) gene of the spodoptera frugiperda, and the agomiR-2c-5p can be used for preventing and treating the spodoptera frugiperda. Therefore, the growth and development of the spodoptera frugiperda larvae are slowed down, the feed intake is reduced, and the death rate is increased. In addition, by adding an analogue agomiR-2c-5p into the feed, the growth of the spodoptera frugiperda can be inhibited, the food intake is reduced, and the death rate is increased. The invention provides a new target and technical support for establishing a new strategy for preventing and controlling the spodoptera frugiperda based on the miRNA.

The application provides a kind of miRNA for targeting ARG1 and its application, belong to gene therapy technical field.The application discloses the application of hsa-miR-4521 for targeting ARG1 in preparation of medicine for preventing and / or treating type 2 diabetes vascular endothelial dysfunction, hsa-miR-4521 can inhibit type 2 diabetes vascular endothelial dysfunction, and circHMGCS1 can be combined hsa-miR-4521 to regulate the expression of target gene ARG1, and then regulate the occurrence of type 2 diabetes vascular endothelial dysfunction, and the medicine related to type 2 diabetes vascular endothelial dysfunction has extensive application prospect, based on the application, the drug or detection kit for treating or preventing the disease related to type 2 diabetes vascular endothelial dysfunction can be prepared.

The invention discloses an SMN gene expression cassette, a virus vector carrying the SMN gene expression cassette and application of the virus vector in treatment of spinal muscular atrophy, and relates to the field of gene therapy. The SMN1 gene in a gene expression cassette is subjected to codon optimization, so that the expression quantity of SMN protein after SMN1 gene transduction is improved, and the clinical dosage is expected to be effectively reduced. Meanwhile, a regulatory element such as a miRNA target sequence is also introduced into the gene expression cassette, so that the immune response of an organism to an adeno-associated virus vector and a transgenic product is relieved, and the safety problem possibly caused by a gene therapy is expected to be reduced.

The present application relates to the technical field of liver drug delivery, and specifically discloses a liver precise expression composition, a vaccine composition and application thereof. The liver precise expression composition comprises: component (1): at least one nucleic acid comprising a nucleotide sequence encoding a target protein and one or more miRNA targeting binding sequences; component (2): a lipid nanoparticle composition which is easy to accumulate in the liver. The liver precise expression composition can be used to prepare a lipid nanoparticle or a vaccine composition, and can be efficiently delivered into the liver and its tissues and cells, and significantly reduces the expression of target genes in non-liver organs, avoiding side effects caused by undesired expression, such as inflammatory reactions or other off-target effects.

A replicating oncolytic virus vector is provided having a modified Enterovirus genome (e.g., a Poliovirus, Coxsackievirus or Echovirus genome), wherein the modified Enterovirus genome has one or more copies of one or more miRNA target sequences inserted into the UTR region (e.g., via substitution) and / or in-frame within the coding region of the Enterovirus genome. Also provided are compositions and methods for treating cancer (including for example, lung cancer).

The invention discloses miR-13b-5p for preventing and treating spodoptera frugiperda and application of the miR-13b-5p, miRNA and miR-13b-5p targeting a key gene for growth and development of the spodoptera frugiperda are screened for the first time, and after an analogue agomiR-13b-5p of the miRNA and miR-13b-5p is injected, the agomiR-13b-5p is found to be capable of inhibiting normal expression of a juvenile hormone epoxy hydrolase (JHEH) gene of the spodoptera frugiperda, so that the growth and development of the spodoptera frugiperda can be inhibited, and the growth and development of the spodoptera frugiperda can be inhibited. Therefore, the growth and development of the spodoptera frugiperda larvae are slowed down, the feed intake is reduced, and the death rate is increased. In addition, by adding an analogue agomiR-13b-5p into the feed, the growth of the spodoptera frugiperda can be inhibited, the feeding amount is reduced, and the death rate is increased. The invention provides a new target and technical support for establishing a new strategy for preventing and controlling the spodoptera frugiperda based on the miRNA.

The invention discloses miR-13a-3p for preventing and treating spodoptera frugiperda and application of the miR-13a-3p, miRNA and miR-13a-3p targeting a key gene for growth and development of the spodoptera frugiperda are screened for the first time, and after an analogue agomiR-13a-3p of the miRNA and miR-13a-3p is injected, the agomiR-13a-3p is found to be capable of inhibiting normal expression of a juvenile hormone esterase (JHE) gene of the spodoptera frugiperda, so that the growth and development of the spodoptera frugiperda can be inhibited, and the growth and development of the spodoptera frugiperda can be inhibited. Therefore, the growth and development of the spodoptera frugiperda larvae are slowed down, the feed intake is reduced, and the death rate is increased. In addition, by adding the analogue agomiR-13a-3p into the feed, the growth of the spodoptera frugiperda can be inhibited, the feeding amount is reduced, and the death rate is increased. The invention provides a new target and technical support for establishing a new strategy for preventing and controlling the spodoptera frugiperda based on the miRNA.

The invention concerns synthetic miRNAs targeting the TOX gene and uses thereof. Pharmaceutical compositions comprising such miRNAs and uses thereof in the medical field, in particular in the treatment of cancer diseases and immunotherapy, are also described.