edicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases and gene therapy (A61K48)

Immunity-inducing means // 2614386
FIELD: biotechnology.SUBSTANCE: invention refers to production of means containing at least one polypeptide selected from SEQ ID NO: 4, 2, 8, 10 and 12, and/or recombinant vector(s), comprising polynucleotide(s) encoding at least one polypeptide, as the active ingredient(s), and can be used in medicine. The resulting means is used for efficient induction of T-cell immunity against malignancies expressing KATNAL1.EFFECT: invention allows to obtain antigen-presenting cells presenting the polypeptide obtained from KATNAL1, and to effectively induce cytotoxic cells against KATNAL1, which is efficient as a therapeutic agent against malignant neoplasms expressing KATNAL1.7 cl, 3 dwg, 3 ex

Reduction of level of lactate and increasing of production of polypeptide by inhibiting expression of lactate dehydrogenase and pyruvate dehydrogenase kinase // 2614125
FIELD: biotechnology.SUBSTANCE: invention relates to biotechnology and concerns method of reducing lactate production in mammalian cells, method of silencing or reduction in mammal cell of transcription of lactate dehydrogenase (LDH) and pyruvate dehydrogenase kinase (PDHK), method of producing mammal cells, which exhibits low production of lactate in culture, vector containing first heterologous nucleotide sequence coding small interfering RNA (siRNA), specific for lactate dehydrogenase, and second heterologous nucleotide sequence coding siRNA specific for pyruvate dehydrogenase kinase, each of which is connected with its promoter.EFFECT: presented inventions allow reducing in cultured cells production of lactate and increasing of production of heterologous polypeptide.40 cl, 6 dwg

Treatment of diseases associated with colonystimulating factor 3 (csf3) by inhibition of natural antisense transcript to csf3 // 2612884
FIELD: biochemistry.SUBSTANCE: invention relates to biochemistry. Invention describes modified oligonucleotides with length from 15 to 30 nucleotides, containing at least one modification, wherein said at least one modification is selected from: at least one modified sugar fragment; at least one modified internucleotide linkage; at least one modified nucleotide; and their combinations. Wherein said oligonucleotides are specifically hybridized with natural antisense CSF3 gene polynucleotide and have sequence at least by 80 % identical to sequence, back complementary section within nucleotides from 1 to 742 of SEQ ID NO: 2, or have sequence at least by 80 % identical to section of sequence SEQ ID NO: 1. Invention also relates to use of such antisense oligonucleotides for treating diseases and disorders associated with expression of CSF3.EFFECT: invention extends range of products for treating diseases and disorders associated with expression of CSF3.30 cl, 1 dwg, 1 tbl, 2 ex

Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof // 2612788
FIELD: medicine.SUBSTANCE: present invention relates to gene therapy for treating eye diseases, more specifically to use vectors for conditional expression of one or more therapeutic protein under the control of gene expression modulation system in the presence of activating ligand in the preparing of a drug, and can be used in medicine.EFFECT: present invention allows to engineer a conditional expression vectors, which can be used for treating macular degeneration or glaucoma in an individual.10 cl, 40 dwg, 8 tbl, 12 ex

Optimized nucleotide sequence and pharmaceutical compositions based thereon with sustained vegf transgene expression // 2612497
FIELD: biotechnology.SUBSTANCE: way to extend the lifetime of vegf transgene mRNA in a mammal cell transfected with the genetic structure. Point deletions of 3'noncoding region of the vascular endothelial growth factor (Vegf) gene are performed, at that, the deleted nucleotide is not replaced or replaced by cytosine in guanine or adenine substitution points. Then, mRNA lifetime is determined for each deletion. Data is analyzed and a genetic structure is designed, comprising 3'noncoding region with aggregated single deletions and/or cytosine substitutions, which showed the best results in terms of mRNA lifetime extension. In addition, versions of plasmid DNA, constructed to implement the said method, and vegf transgene mRNA obtained by plasmid DNA transcription, are proposed. The invention also relates to plasmid DNA aplication, including its use as part of a pharmaceutical composition for tissue regeneration.EFFECT: invention allows to increase overall therapeutic vegf protein production due to an increased mRNA lifetime achieved by introduction of optimal changes in the vegf gene 3'UTR sequence.8 cl, 9 dwg, 2 tbl, 2 ex

Treatment of pancreatic developmental gene related diseases by inhibition of natural antisense transcript to pancreatic developmental gene // 2612161
FIELD: chemistry.SUBSTANCE: present invention relates to antisense oligonucleotides, modulating expression of pancreatic developmental gene, in particular, by targeted interaction with natural antisense polynucleotides of pancreatic developmental gene. Said oligonucleotides have a length from 15 to 30 nucleotides, have a sequence at least 90 % identical to sequence, inverse to complementary section from 1 to 1 235 nucleotide sequence SEQ ID SEQ ID NO: 6, from 1 to 17 964 nucleotide sequence SEQ ID NO: 7, from 1 to 50 003 nucleotide sequence SEQ ID SEQ ID NO: 8, from 1 to 486 nucleotide sequence SEQ ID NO: 9, from 1 to 494 nucleotide sequence SEQ ID NO: 10, from 1 to 1 992 nucleotide sequence SEQ ID NO: 11, or from 1 to 1 767 nucleotide sequence SEQ ID NO: 12, or have a sequence, at least 90 % identical to section of sequence selected from SEQ ID NOS: 1-5, and are specifically hybridised with natural antisense polynucleotide, selected from SEQ ID NOS: 6-12, wherein said oligonucleotides can optionally contain one or more modifications, selected from following: at least one modified sugar fragment, at least one modified inter-nucleoside bond, at least one modified nucleotide and combination thereof. Present invention also relates to use of such antisense oligonucleotides for treating diseases and disorders, associated with expression of pancreatic developmental genes.EFFECT: invention widens range of products for treating diseases and disorders associated with expression of pancreatic developmental genes.31 cl, 5 dwg, 1 tbl, 2 ex

Virions of adeno-associated virus with optional capsid and methods of their use // 2611202
FIELD: medicine; biotechnology.SUBSTANCE: invention relates to biotechnology, virology, and medicine. Virions of adeno-associated virus (AAV) with optional capsid protein are presented, where AAV virions show high infection load of retinal cell at intravitreal injection compared with wild type AAV. Methods of delivering gene product in retinal cell of person and methods of treating eye diseases are also described.EFFECT: virions of adeno-associated virus with optional capsid and methods for using them are presented.24 cl, 25 dwg, 2 tbl, 3 ex

Treatment of rnase h1 related diseases by inhibition of natural antisense transcript to rnase h1 // 2611192
FIELD: biotechnology.SUBSTANCE: oligonucleotides are discribed that enhance RNase H1 gene expression via targeted contact with natural antisense polynucleotides RNase H1.The present invention also relates to the use of the described oligonucleotides for treatment of diseases and disorders associated with RNase H1 expression.EFFECT: creation of additional means for treatment of diseases and disorders associated with RNase H1 expression.30 cl, 1 dwg, 2 ex

Treatment of diseases, associated with sex hormones binding globulin (shbg), by inhibition of natural antisense transcript to shbg // 2611191
FIELD: biochemistry.SUBSTANCE: invention relates to biochemistry. Invention describes antisense oligonucleotides, modulating expression of sex hormones binding globulin (SHBG), in particular by targeted interaction with natural antisense polynucleotides of sex hormones binding globulin (SHBG). Present invention also relates to use of such antisense oligonucleotides for treating diseases and disorders, associated with SHBG expression. Said oligonucleotides have length from 19 to 30 nucleotides, wherein they are specifically hybridized with natural antisense polynucleotide of SHBG gene and have sequence identical at least by 90 % to sequence reverse complementary to section within from 1 to 3016 nucleotide of sequence SEQ ID NO: 2 or within from 1 to 1609 nucleotide of sequence SEQ ID NO: 3, or have sequence, identical at least by 90 % to section of sequence SEQ ID NO: 1, wherein they can optionally contain one or more modifications, selected from following: at least one modified sugar fragment, at least one modified internucleoside link, at least one modified nucleotide and their combinations.EFFECT: invention extends range of products for treating diseases and disorders, associated with SHBG expression.32 cl, 1 dwg, 2 ex

Treatment of diseases related with gene dlg by inhibition of natural antisense transcript of dlg gene // 2611190
FIELD: biochemistry.SUBSTANCE: invention relates to biochemistry. Described an oligonucleotide with length approximately from 15 to 30 nucleotides, containing at least one modification, note here that said at least one modification selected from: at least one modified sugar residue; at least one modified internucleotide linkage; at least one modified nucleotide; and their combinations. Note here that said oligonucleotide is hybridized with natural antisense transcript of gene Discs large homolog 1 (DLG1) and has the sequence, at least at 80 % identical to reverse component of section of the sequence SEQ ID NO: 2, or has the sequence, at least at 80 % identical to the section of the sequence SEQ ID NO: 1. Invention also relates to use of said antisense oligonucleotide for treatment diseases and disorders associated with expression of gene DLG1.EFFECT: invention extends the range of agents for treatment diseases and disorders associated with DLG gene expression.33 cl, 2 dwg, 1 tbl, 3 ex

Treatment diseases, associated with interferon-regulatory factor 8 (irf8), by inhibition of natural antisense transcript to irf8 // 2611187
FIELD: chemistry.SUBSTANCE: present invention relates to antisense oligonucleotides, modulating expression and/or function of interferon-regulatory factor 8 (IRF8), in particular, by targeted interaction with natural antisense polynucleotides of interferon regulatory factor 8 (IRF8).EFFECT: present invention also relates to identification of such antisense oligonucleotides and use thereof in treating diseases and disorders associated with expression of IRF8.32 cl, 1 dwg

Treatment of tumor protein 63 (p63) related diseases by inhibition of natural antisense transcript to p63 // 2611186
FIELD: biochemistry.SUBSTANCE: invention relates to biochemistry. Described oligonucleotides with length from 15 to 30 nucleotides, containing at least one modification, note here that said at least one modification selected from: at least one modified sugar fragment; at least one modified internucleotide linkage; at least one modified nucleotide; and their combinations. Note here that said oligonucleotides are specifically hybridized with natural antisense polynucleotide of gene p63, increasing such way gene expression of tumor protein 63 (p63) in vivo or in vitro, and have sequence at least at 90% identical to sequence, reverse complementary to a section within from 1 to 288 nucleotide sequence SEQ ID NO: 2, or have sequence, at least at 90% identical to a section of sequence SEQ ID NO: 1. Present invention also relates to use of such antisense oligonucleotides for treatment of diseases and disorders associated with expression of p63.EFFECT: invention extends the range of products for treating diseases and disorders associated with expression of p63.33 cl, 1 dwg, 2 ex

Treatment of fibroblast growth factor 21 (fgf21) related diseases by inhibition of natural antisense transcript to fgf21 // 2610661
FIELD: biotechnology.SUBSTANCE: described oligonucleotides, that increase expression of a gene of fibroblast growth factor 21 (FGF21), by interaction with the natural targeting antisense polynucleotides fibroblast growth factor 21 (FGF21). The present invention also relates to the use of the described oligonucleotides for the treatment of diseases and disorders associated with the expression of FGF21.EFFECT: invention expands the arsenal of tools aimed for the treatment of diseases and disorders associated with the expression of FGF21.26 cl, 2 dwg, 3 ex, 1 tbl

Treatment of diseases, associated with hepatocyte growth factor (hgf), by inhibition of natural antisense transcript to hgf // 2609631
FIELD: biochemistry.SUBSTANCE: invention relates to biochemistry. Invention describes oligonucleotides, modulating expression of hepatocyte growth factor (HGF), in particular, by means of targeted interaction with natural antisense polynucleotides of hepatocyte growth factor (HGF). Present invention also relates to the use of mentioned oligonucleotides for treating diseases and disorders, associated with HGF expression.EFFECT: invention extends range of products for treating diseases and disorders, associated with HGF expression.32 cl, 1 dwg, 2 ex

ethods and compositions for treating hemophilia b // 2608643
FIELD: biochemistry.SUBSTANCE: described protein which binds to genome factor IX (FIX), polynucleotide coding such protein, recovered host cell expressing the protein and method for protein expression. Presented protein includes genetically engineered DNA-binding domain of protein of "zinc finger" type, where DNA-binding domain contains four or five regions of recognising"zinc finger".EFFECT: presented inventions can be used in clinical practice in patients with hemophilia B.15 cl, 5 dwg, 1 tbl, 5 ex

Treating diseases, associated with nanog, by inhibition of natural antisense nanog transcript // 2608493
FIELD: biochemistry.SUBSTANCE: invention relates to biochemistry. Methods of increasing NANOG polynucleotide expression with help of oligonucleotides with length from 19 to 30 nucleotides are disclosed. Corresponding oligonucleotides and composition, containing them, are described.EFFECT: method of preventing or treating diseases, associated with at least one NANOG polynucleotide and/or at least one product, coded by said polynucleotide is also described, including: administering to patient of therapeutically effective dose of at least one described oligonucleotide.35 cl, 1 dwg, 1 tbl, 2 ex

Serotonin 5-ht3 receptor antagonists for application in treatment of lesional vestibular disorders // 2608458
FIELD: medicine.SUBSTANCE: invention relates to medicine and consists in application of serotonin 5-HT3 receptor antagonist for treatment of damages during vestibular disorders, wherein, mentioned damage is characterized by damage of internal ear cells and/or vestibular nerve cells, wherein, serotonin 5-HT3 receptor antagonist is selected from a group comprising ondansetron, palonosetron, tropisetron, lerisetron, alosetron, granisetron, dolasetron, bernesetron, ramosetron, azasetron, itasetron, zakoprid and cilansetron; and mentioned serotonin 5-HT3 receptor antagonist is introduced to the patient, at least during 5 days.EFFECT: treatment of damages during vestibular disorders.4 cl, 4 ex, 6 dwg

Novel viral vector construct for neuron specific continuous dopa synthesis in vivo // 2606012
FIELD: medicine.SUBSTANCE: inventions relate to dicistronic expression vector, its application for amelioration of Parkinson's disease, pharmaceutical composition and a method for determining the expression ratio of polypeptide of GTP-cyclohydrolase 1 and polypeptide of tyrosine hydroxylase expressed by said dicistronic vector. Presented vector contains the first top expression cassette along the transcription and the second lower expression cassette along the transcription. Said first top expression cassette along the transcription contains nucleotide sequence containing the first promoter sequence functionally connected with the first nucleotide sequence, wherein said first nucleotide sequence encodes GTP-cyclohydrolase 1 polypeptide (GCH1; EC 3.5.4.16). Said second lower expression cassette along the transcription contains nucleotide sequence containing the second promoter sequence functionally connected with the second nucleotide sequence. Said second nucleotide sequence encodes tyrosine hydroxylase polypeptide (TH; EC 1.14.16.2). At that, the vector is adeno-associated vector (AAV) and said second nucleotide sequence is functionally related to woodchuck posttranscriptional regulatory element (WPRE). Said first and second promoters are promoters of synapsin 1, the first top expression cassette along the transcription and second lower expression cassette along the transcription contain polyadenylation sequence, and expression cassettes of the dicistronic expression vector contain 5'-end repeated sequence and 3'-end repeated sequence. Polypeptides of tyrosine hydroxylase and GTP-cyclohydrolase 1 can be expressed in ratio from 3:1 to 7:1, respectively. Inventions can be used for recovery of catecholamine balance in a subject in need thereof.EFFECT: present invention is applicable for treating disorders associated with dopamine deficiency.21 cl, 8 dwg, 1 tbl, 3 ex

ethod for screening substances having weight control effect // 2603745
FIELD: biotechnology.SUBSTANCE: invention relates to genetic engineering, particularly, to screening of substances having weight control effect, and can be used in medicine. Method of screening substances having weight control effect, involves bringing test substance into contact with cells expressing Synoviolin gene, and identifying whether above test substance has or not inhibiting effect on Synoviolin gene expression. Pharmaceutical compositions include nucleic acid, which suppresses Synoviolin gene expression, or Synoviolin protein ubiquitinylation activity inhibitor.EFFECT: invention makes it possible to effect on weight reduction by inhibition of gene expression or Synoviolin protein activity.4 cl, 5 dwg, 1 ex

Particulate substances comprising ceramic particles for delivery of biomolecules // 2600841
FIELD: medicine.SUBSTANCE: series of inventions relates to medicine. A particulate substance is described that comprising: particles of a ceramic matrix bearing a functional group, which is capable of promoting penetration of the particles into cells; and a biomolecule disposed within pores of the particles, where said biomolecule being releasable from the particles by dissolution of the ceramic matrix.EFFECT: agent provides good penetration of the active substance in target cells.56 cl, 22 dwg

ethod for gene therapy treatment of diabetic foot syndrome // 2599507
FIELD: medicine.SUBSTANCE: present group of inventions relates to medicine. Disclosed is the use of pCMV-VEGF plasmid splicing version 165 (Seq # 1) for treating diabetic foot syndrome and method for treating diabetic foot syndrome.EFFECT: presented group of inventions provides improved treatment results for diabetic foot by means of gene therapy.3 cl, 3 dwg, 4 tbl

Tissue-regeneration promoter using recruitment of bone marrow mesenchymal stem cells and/or pluripotent stem cells in blood // 2599448
FIELD: medicine.SUBSTANCE: presented inventions relate to use of an agent stimulating tissue regeneration, and a method of stimulating repair of mesenchymal or epithelial neurological tissues by administering said agent. Described agent is an S100A8 protein, a cell which secretes an S100A8 protein, a vector into which DNA encoding S100A8 protein is inserted, an S100A9 protein, a cell which secretes an S100A9 protein or a vector into which DNA encoding an S100A8-9 protein is inserted.EFFECT: presented inventions can be used for inducing healing of injured tissues by recruiting bone-marrow-derived cells in area of damage, thereby treating such diseases as extensive pitting skin, bone fractures, cerebral infarction.7 cl, 44 dwg, 1 tbl, 9 ex

Use of structure for dna expression // 2598713
FIELD: medicine; biotechnology.SUBSTANCE: disclosed is use of a dumbbell-shaped linear, covalently closed DNA expression construct with a double-stranded stem and single-stranded loops located at both ends of stem, wherein stem of complementary deoxyribonucleic acids of a circular DNA strand comprises a promotor sequence, a coding sequence and a termination signal, where DNA construct codes TNF-α, for treating melanoma, wherein said DNA construct is administered by jet injection, and said construct is administered simultaneously or successively with vindesine.EFFECT: use of said DNA construct significantly enhances vindesine action.5 cl, 7 dwg, 1 tbl

odulation of antigen immunogenicity by deleting epitopes recognized by nkt-cells // 2598247
FIELD: biotechnology.SUBSTANCE: invention relates to biotechnology, specifically to prevention of undesired immune responses in mammals, and can be used in medicine. Method consists in (a) establishing at least one CD1d-binding motif of NKT cell epitope in peptide or polypeptide, wherein said epitope comprises the motif of [FWTHY]-X2X3-[ILMV]-X5X6-[FWTHY], wherein X2, X3, X5, X6 is any aminoacid, (b) removing said epitope by replacement of aminoacid residues in position P1 and/or P7 on non-hydrophobic residues; and (c) obtaining isolated peptide or a polypeptide with decreased ability to activate NKT cells in mammal.EFFECT: invention allows to reduce undesirable immune responses in mammals towards allofactors, towards viral vectors used for gene therapy and gene vaccination, towards proteins to which subjects are naturally exposed, towards genetically-modified organisms and towards undesirable effects related to vaccine administration for allergic or infectious diseases.10 cl, 3 dwg, 5 ex

Conjugates and compositions for immunotherapy and anticancer treatment // 2597989
FIELD: biotechnology.SUBSTANCE: invention refers to immunology, and can be used in medicine. Composition based on combined application of ApoA, interleukin 15 and Sushi domain alpha chain of IL15 receptor, is used for anti-tumour immune response in individual.EFFECT: use of ApoA1 together with IL15 and Sushi IL15ra domain provides synergetic effect on stimulating anti-tumour immune response in mammal by intensifying expansion of anticancer CTL.18 cl, 13 dwg, 13 ex

ethod for producing regulatory dendritic cells // 2597976
FIELD: biotechnology.SUBSTANCE: invention relates to biotechnology, specifically to obtaining regulatory dendritic cells, and can be used in medicine for treating diseases due to immunological anomalies and excess immune responses. Method comprises differentiation into regulatory dendritic cells, which can be induced in order to obtain regulatory dendritic cells, in presence of (S)-(+)-1-(5-hydroxy-1,5-dimethylhexyl)-3-[7-(4-methoxyphenyl)-[1,2,4]triazolo[1,5-a]pyrimidin-2-yl]urea, GM-CSF and IL-4 and subsequent ageing of regulatory dendritic cells in presence of TNF-α and/or LPS.EFFECT: higher quality and longer period of survival of patients during transplantation of said regulatory dendritic cells.10 cl, 2 dwg, 2 tbl, 4 ex

Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua // 2597972
FIELD: biotechnology.SUBSTANCE: invention relates to biotechnology, specifically to a method of increasing polynucleotide expression of alpha-L-Iduronidase (IDUA), and can be used in medicine. Invention also relates to identification of antisense oligonucleotides, which are targeted on natural antisense IDUA polynucleotide sequences chosen from SEQ ID NO: 2-9.EFFECT: invention increases polynucleotide expression of IDUA, which can be used for treating diseases and disorders associated with IDUA gene expression.39 cl, 5 dwg, 7 ex

Vectors and sequences for treating diseases // 2588667
FIELD: biochemistry.SUBSTANCE: described is nucleic acid molecule for gene therapy of mucopolysaccharidoses. Also described are vectors and pharmaceutical compositions for treating mucopolysaccharidosis disease.EFFECT: invention widens range of agents for treating mucopolysaccharidoses.15 cl, 15 dwg, 1 tbl, 11 ex

Novel peptides with analgesic effect, inhibiting asic-rfyfks // 2583299
FIELD: biotechnologies.SUBSTANCE: present invention relates to biotechnology, specifically to obtaining new recovered peptides, which cause analgesia and inhibit ASIC channels (proton-sensitive ion channels), to polynucleotides coding said peptides, as well as vectors and their application, and can be used in medicine. Above peptides allocated from poison of Dendroaspis polylepis snake and have amino acid sequence of LKCX4QHGKVVTCHRDMKFCYHNTGMPFRNLKLILQGCSSSCSETENNKCCSTDRCNK, wherein X4 denotes any amino acid; or sequence with similarity of at least 56 % with above sequence and preserving analgesic activity. EFFECT: invention allows to produce peptide inhibiting ASIC channels, containing at least one subunit, selected from group consisting of subunits ASIC1a and ASIC1b.14 cl, 7 dwg, 1 tbl, 7 ex

Low-molecular conjugates for intracellular delivery of nucleic acids // 2582235
FIELD: chemistry.SUBSTANCE: disclosed are compounds and compositions based thereon, used in medicine, of formulae and, a nucleic acid (II), where Y is -(CH2)3-; R1 is -(C1-6)alkyl; or -(CH2)m-phenyl, optionally substituted up to four times with a substitute selected from: -NO2, -CN, or a halogen; R2 is hydrogen; -(CH2)k-phenyl; -(C1-6)alkyl; -(CH2)k-C(O)-NH2; or -(CH2)k-N-C(Ph)3. The phenyl rings are optionally substituted with -O-(C1-4)alkyl; R3 is -NH-phenyl, wherein the phenyl is further substituted with a substitute independently selected from -(CH2)-OH or -(CH2)-O-C(O)-O-(4-nitrophenyl); k equals 1, 2, 3, 4, 5 or 6; m equals 1, 2, 3 or 4; and n equals 0 or 1, Ra is -(CH2)k-NH2; R1, wherein the nucleic acid is a single-stranded RNA oligonucleotide.EFFECT: novel compounds for nucleic acid delivery in cells and compounds for production thereof.10 cl, 653 ex, 19 tbl, 6 dwg

ethod of inducing cytotoxic anti-tumour immune response in vitro with using dendritic cells, transfected by rna tumour cells, to non-small cell lung cancer // 2578008
FIELD: biotechnologies.SUBSTANCE: invention can be used for inducing an antitumour immune response in vitro. Method involves production of adhesive and non-adhesive fractions of mononuclear cells (MNC) recovered from patient's peripheral blood, combined cultivation recovered from patient's peripheral blood MNC non-adhesive fraction with mature dendritic cells (DC), made from adhesive MNC fraction, transfected with RNA of tumour cells. Then, DC is subjected to stimulated ageing. Combined cultivation non-adhesive MNC fraction and transfected RNA autologous tumour cell DC is performed for 5 days. Note here that for producing mature DC after three days of cultivation of adhesive MNC fraction are transfected immature DC tumour RNA, and one day after transfection immature DC is for days stimulated ageing by adding a culture of immature DC tumour necrosis factor-α (TNF-α) and Interleukin-1β (IL-1β) in the following proportions: TNF-α : IL-1β= 2.5:1.EFFECT: invention allows to effectively induce cytotoxic anticancer immune response to non-small cell lung cancer in vitro with the help of dendritic cells, transfected by RNA tumour cells.1 cl, 1 tbl

ethod of generating cytotoxic cells with activity against non-small cell lung cancer cells // 2577992
FIELD: biotechnologies.SUBSTANCE: invention relates to biotechnology, particularly to immunology, and can be used for generation of cytotoxic cells with activity against non-small cell lung cancer. Method involves combined cultivation in presence of recombinant human interleukine-12 and recombinant human interleukin-18, non-adhesive fraction of mononuclear cells (MNC) recovered from peripheral blood of patient with non-small cell lung cancer with dendritic cells obtained from monocytes of adhesive MNC fraction. To produce mature DC after three days of cultivation of adhesive MNC fraction, obtained immature DC are first co-cultivated for one day with tumour cell lysate non-small cell lung cancer with subsequent addition of TNF-α. During one day after loading lysate simultaneously with addition of proinflammatory cytokine TNF-α cytokine IL-1 is addedβ and matured loaded with lysate in presence of both cytokines.EFFECT: higher efficiency of generation of cytotoxic cells with activity against non-small cell lung cancer.1 cl, 1 tbl

System for stimulation of genes expression and vector containing said system // 2577971
FIELD: bioengineering.SUBSTANCE: claimed invention discloses a gene expression cassette. The latter contains the DNA-construct where the promoter, the gene, that should be expressed, and the sequence of poly A addition are bonded in such order. Besides, it contains the enhancer(s) or enhancer(s) with UAS doped with its part upstream of its transcription. The latter include at least one enhancer hTERT wherein the said enhancers with UAS are doped directly downstream of transcription from the sequence of poly A addition. The gene expression cassette is used in the procedures of gene expression and protein production coded by this gene as well as in preparations for the detection or treatment of diseases.EFFECT: increased expression of protein from the gene whereat the enhancer is added upstream of transcription from the promoter.18 cl, 75 dwg, 10 ex

ethod for identifying compounds for treating cancer // 2575828
FIELD: medicine, pharmaceutics.SUBSTANCE: invention refers to oncology and concerns a complex containing a combination of polyinosinic-polycytidylic acid (pIC) and polyethylenimine (PEI), and using it as an agent for treating cancer, particularly melanoma.EFFECT: invention contains a pharmaceutical composition containing the complex applicable in treating melanoma, wherein the above complex induces autophagia in melanoma cells or in a cell line produced from the melanoma cells, and the above complex contains the combination of pIC and linear PEI in N/P ratio 1:5.10 cl, 1 tbl, 13 ex, 15 dwg

Obtaining of complexes of nucleic acids and cationic components cross sewed by disulfide bonds intended for transfection and immunostimulation // 2575603
FIELD: biotechnologies.SUBSTANCE: complex contains the polymeric carrier formed by the cationic components which are cross sewed by disulfide bonds where the cationic components cross sewed by disulfide bonds are cationic peptides, and the named disulfide bonds are formed by the cysteine residues which are comprised by cationic peptides localized near the terminal ends of cationic peptides, and the cargo molecule which at least one one-chained molecule of RNA.EFFECT: invention allows to perform efficient transfection of cells by nucleic acids both in vivo, and in vitro, and is intended for induction of congenital or adaptive immune response.10 cl, 15 dwg, 1 tbl, 1 ex

Pharmacological combination of polycationic carrier peg-pei-tat with included in it plasmid carrying therapeutic genes hsvtk and gm-csf for gene therapy of tumorous diseases // 2575077
FIELD: bioengineering.SUBSTANCE: medical anticancer drug is produced, it contains the gene structure in the form of a plasmide DNA, comprising genes of the thymidine kinase of the herpes simplex virus (HSVtk), and a granulocytic-macrophage colony-stimulating factor (GM-CSF), included in a polymer carrier - copolymer PEG-PEI-TAT peptide. Wherein the gene structure ensures the expression of both therapeutic genes HSVtk and GM-CSF from the same vector. The gene structure is intended for intratumoural introduction under the mode of the progressively increased single dose calculated depending on the tumour size, with the further injection of the prodrug - ganciclovir.EFFECT: invention ensures the effective delivery and diffusion in the tumour cells of the plasmid pCMV-HSVtk-GM-CSF, and ensures with high efficiency the selective destruction of cancer cells preventing further metastasis.8 cl, 9 dwg, 16 tbl, 7 ex

ethod for correction of age-related pathological skin conditions // 2574905
FIELD: medicine.SUBSTANCE: invention concerns a method for correcting individual's age-related pathological skin conditions; the method involves using patient's autogenous fibroblasts and administering them to the patient with material sampling and cell growth followed by patient's fibroblast culture recovery; the fibroblast culture is genetically analysed by detecting DNA sequence and activity of genes specified in a group consisting of TGFB1, TGFBR2, COL1A1, COL1A2, SOD1, SOD2, GPX1, GPX3, CLCA2; that is followed by carrying out a comparative analysis of the obtained results in relation to normal DNA sequences and normal expressions of respective genes and creating genetic constructs containing complementary DNA of those genes, activity of which is changed in a patients or DNA structure of which is irregular, and introducing these genetic constructs into the patient's fibroblast culture, and administering these modified autogenous fibroblasts into the patient.EFFECT: reducing the clinical signs of skin ageing accompanied by positive progression of skin functional parameters.8 cl, 16 ex, 13 dwg, 20 tbl
Photosensitising compositions // 2574019
FIELD: chemistry.SUBSTANCE: invention relates to photochemotherapy and photodynamic therapy, namely to application of pharmaceutically acceptable salt of amphiphilic photosensitising preparation in method of photochemical internalisation, where said salt possesses water solubility of at least 30 mg/ml and is selected from diethanolamine salt TPCS2a, diethanolamine salt TPPS2a, ethanolamine salt TPPS2a and triethanolamine salt TPPS2a.EFFECT: invention provides improvement of method for carrying out photochemical internalisation.15 cl, 22 ex

Constructed cells, expressing multiple immunomodulators, and thereof application // 2573912
FIELD: chemistry.SUBSTANCE: claimed invention relates to field of biotechnology and can be used for recombinant expression of immunomodulatory proteins. Vector, containing polynucleotide which codes gene switch, is constructed, with said polynucleotide containing (1) at least one transcription factor sequence which is functionally bound with promoter, with said at least one transcription factor sequence coding ligand-dependent transcription factor, and (2) polynucleotide, which codes peptide IL-12 an one or more immunomodulatory polypeptides, selected from IL-2, IL-7, IL-15, IL-18, IL-21, GM-CSF, CCL3 (MIP-1a), CCL5 (RANTES), CCL7 (MCP3), XCL1 (lymphotactin), CCL19 (MIP-3b), CXCL9 (MIG), CXCL10 (IP-10), CXCL12 (SDF-1), CCL21 (6Ckine) or TNF-alpha.EFFECT: invention makes it possible to use vector for controlled expression of immunomodulatory proteins.12 cl, 9 dwg, 4 tbl, 2 ex

Lipid particles containing nucleic acids and related methods // 2573409
FIELD: chemistry.SUBSTANCE: group of inventions relates to biochemistry. Claimed are: lipid particle for delivery of nucleic acid (versions), method of introducing nucleic acid in cell, method of producing lipid particles, which include nucleic acid. Lipid particle contains core, consisting of nucleic acid and cation lipid, and secondary lipids, surrounding said core. In other version lipid particle contains core, consisting of nucleic acid, cation lipid and secondary lipids, and secondary lipids, surrounding said core. Method of introduction of nucleic acid into cell includes bringing cell in contact with said lipid particles. Method of producing lipid particles, containing nucleic acid, includes introduction of first flow, which contains nucleic acid, and introduction of second flow, which contains substances, forming lipid particle, into device providing flowing of first and second flows, flowing first and second flows from first part of device into second part of device, mixing flows in second part of device to provide third flow, containing lipid particles.EFFECT: inventions provide improvement of lipid particles, containing therapeutic substance, increased efficiency of encapsulation of nucleic acids into lipid particle.18 cl, 33 dwg, 1 tbl, 5 ex

Treatment of disease, disorders or pathological conditions of lungs with application of placental cells // 2570550
FIELD: medicine.SUBSTANCE: invention relates to the field of molecular biology. Claimed application of a therapeutically effective quantity of placental stem cells in obtaining a pharmaceutical composition for application in the treatment of an individual, who has a disease, disorder or pathological condition of the lungs, with the therapeutically effective quantity representing the quantity, sufficient to cause a detectable improvement of one or several symptoms of the said disease, disorder or pathological condition; where the said disease, disorder or pathological condition of the lung represents pulmonary sarcoidosis, asthma, bronchitis or acute respiratory distress-syndrome, and the placental stem cells are CD10+, CD34-, CD105+ and CD200+, which is determined by means of flow cytometry.EFFECT: obtaining the pharmaceutical composition for application in the treatment of an individual, who has a disease, disorder or pathological condition of the lungs.12 cl, 4 ex, 2 tbl

Introduction of anti-coagulating system reg1 // 2568579
FIELD: medicine, pharmaceutics.SUBSTANCE: invention relates to medicine and can be used for the introduction of an anti-coagulating system to a subject requiring it, where the anti-coagulating system includes an aptamer, binding IX/IXa factor, and an aptamer-binding antidote. For this purpose carried out are: a) measurement of the subject's weight in kilograms; b) introduction to the subject of an aptamer dose, effective for the achievement of coagulation inhibition in the subject, where the aptamer includes sequence SEQ ID No:1 and where the aptamer dose constitutes from 0.1 mg/kg to 2.0 mg/kg or from 5 mg/kg to 10 mg/kg; and c) introduction of the aptamer antidote dose to the subject, where the antidote includes sequence SEQ ID No:2, and the antidote dose is based only on the wt/wt ratio with the aptamer dose, and where the wt/wt ratio of the aptamer dose with the antidote dose constitutes from approximately 0.1:1 to approximately 20:1, and where the subject is subjected to procedures of re-vascularisation of peripheral vessels.EFFECT: neutralisation of the aptamer activity to a desirable degree is provided, with the antidote dose being fully based on its connection with the aptamer dose.17 cl, 24 dwg, 7 tbl

Interferon beta immunobiological agent for bladder cancer therapy and method for using same // 2568575
FIELD: medicine.SUBSTANCE: group of inventions refers to an immunobiological agent for bladder cancer treatment on the basis of an adenoviral vector containing a promoter-controlled interferon beta gene, wherein the above adenoviral vector is human adenovirus; the agent additionally contains an enzyme, which provides bladder mucosa secretion splitting. The group of inventions also concerns a method for using the immunobiological agent for bladder cancer treatment involving administering the above immunobiological agent into the bladder.EFFECT: prolonged action of the immunological agent.7 cl, 5 ex, 5 dwg, 1 tbl

Compositions and methods of modulating smn2 splicing in subject // 2566724
FIELD: medicine, pharmaceutics.SUBSTANCE: claimed group of inventions relates to field of medicine. Claimed is method of relieving at least one symptom of spinal muscular atrophy in subject, which includes introduction to subject of antisense compound, containing antisense oligonucleotide, complementary to intron 7 of pre-mRNA, coding human SMN2. Claimed is application of such antisense compound in production of medication for relieving or treatment of at least one symptom of spinal muscular atrophy in subject.EFFECT: claimed group of inventions provides effective means and methods for relieving spinal muscular atrophy in subject.13 cl, 13 dwg, 14 tbl, 14 ex

Improved composition for inhibition of tumour cell proliferation // 2565542
FIELD: medicine, pharmaceutics.SUBSTANCE: invention relates to field of biotechnology, in particular to obtaining stimulated dendritic cells (DC), and can be applied in medicine for immunotherapy of cancer. Mature pro-inflammatory DC is obtained by ex vivo induction of its maturation by processing immature DC with such substances as sodium salt of polyynosinic-polycytidinic acid (poly-Y:C), resiquimod (R848) and interferon-gamma (IFN-g), but not prostaglandin E2 (PGE2). Obtained DC is applied in treatment of cancer in individual by injection into tumour. Said pro-inflammatory mature DC is allogenic with respect to said individual.EFFECT: invention makes it possible to efficiently inhibit proliferation of tumour cells.14 cl, 14 dwg, 6 tbl

ethod of obtaining pegylated oligonucleotides // 2564855
FIELD: chemistry.SUBSTANCE: claimed invention relates to method of obtaining pegylated oligonucleotide, which can be used for obtaining biologically active substances. Method includes: (a) synthesis of non-pegylated oligonucleotide on solid carrier, (b) cleavage of non-pegylated oligonucleotide from solid carrier and removal of protective groups from oligonucleotide, (c) desalination of non-pegylated oligonucleotide with application of ultrafiltration, (d) pegylation of non-pegylated oligonucleotide to obtain pegylated oligonucleotide, (e) purification of pegylated oligonucleotide with application of anion-exchange HPLC, (f) desalination and additional purification of pegylated oligonucleotide with application of ultrafiltering membrane with threshold of molecular weight from 10 kDa to 30 kDa, where ion-exchange purification of non-pegylated oligonucleotide is not realised between stages (b) and (c).EFFECT: claimed method is characterised by improved efficiency.10 cl, 10 dwg, 11 tbl, 2 ex

Composition for treating arthritis // 2563360
FIELD: medicine, pharmaceutics.SUBSTANCE: invention refers to biotechnology, more specifically to producing a population of collagen II-specific Tr1-cells, and can be used in medicine. The population of collagen II-specific Tr1-cells with the quiescent phenotype of CD4+CD25-FoxP3- is recovered. The produced population of Tr1-cells combined with one or more pharmaceutically acceptable carriers are used as an ingredient of a pharmaceutical composition for treating arthritis.EFFECT: invention enables producing the effective agent for treating arthritis.12 cl, 6 dwg

Fgf-r-fc fused protein and use thereof // 2560573
FIELD: chemistry.SUBSTANCE: invention relates to biotechnology, specifically to fibroblast growth factor receptor (FGFR) hybrid proteins and can be used in medicine to treat diseases associated with excessive FGF expression. A soluble FGFR fused protein is constructed, which consists of a fragment derived from an intermediate functional sequence (IFS) of a FGFR Ig-like domain, a second FGFR Ig-like domain (D2), a third FGFR Ig-like domain (D3) and an immunoglobulin Fc region. The obtained fused protein is used to inhibit angiogenesis in mammals.EFFECT: invention increases affinity of binding with FGF.11 cl, 6 dwg, 3 tbl, 7 ex

Sirna conjugate and method for producing it // 2558258
FIELD: medicine, pharmaceutics.SUBSTANCE: present invention refers to biotechnology and represents a conjugate used for siRNA intracellular delivery and containing siRNA, which is conjugated by a covalent bond with a hydrophilic compound on one side, e.g. PEG, and with a hydrophobic compound, e.g. cholesterol, on the other side. By self-assembly, the conjugates are able to form homogenous nanoparticles, micellas, wherein the hydrophobic compounds are packed inside the micella; siRNA - between the hydrophobic and hydrophilic compounds, and the hydrophilic compounds - outside. The present invention also discloses methods for producing the above conjugate, pharmaceutical compositions containing the above nanoparticles for the gene therapy of various diseases depending on specific siRNA delivered. What is also disclosed is a pharmaceutical composition for treating cancer, which contains the nanoparticles containing survivin-specific siRNA.EFFECT: invention enables increasing the siRNA stability in a living body, providing thereby the effective delivery of therapeutic siRNA into cells and the shown activity of siRNA even in a low dose of a relatively low concentration.25 cl, 19 dwg, 1 tbl, 6 ex

Oligodeoxyribonucleotide inhibitor of human dna-methyltransferase 1 // 2553349
FIELD: biotechnology.SUBSTANCE: oligodeoxyribonucleotide inhibitor of human DNA-methyltransferase 1 (Dnmt1) is provided, characterized in that it has a self-complementary structure, which forms a double-stranded pin, has an unpaired CA pair in the recognition site of the enzyme of human DNA-methyltransferase 1 and comprises in its composition 5-methylcytosine (5mC) and thiophosphates instead of phosphates.EFFECT: invention provides selective inhibition of the Dnmt1 enzyme activity in reactions in vitro and in vivo.3 dwg, 3 ex
 
2551335.
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