Abstract: An object of the present invention is to provide a vector capable of oligodendrocyte-specifically suppressing expression of the PLP1 gene for treating PMD caused by abnormality of the PLP1 gene, and a promoter and miRNA therefor, and a pharmaceutical composition comprising the vector. The oligodendrocyte-specific promoter of the present invention comprises a nucleic acid having a sequence identity of at least 90% to a nucleotide sequence set forth in SEQ ID NO: 1. The miRNA of the present invention specific to the PLP1 gene comprises a pair of nucleotide sequences consisting of a specific antisense sequence and sense sequence.

Abstract: The present invention provides a compound having a particular chemical structure or a pharmacologically acceptable salt thereof which has an excellent inhibitory effect on EZH1 and/or EZH2 activity. The present invention provides a compound having a 1, 3-benzodioxole structure represented by the general formula (I) or a pharmacologically acceptable salt thereof, or a pharmaceutical composition comprising the compound (wherein R1, R2, R3, R4, R5, R6, and V in the formula (I) are each as defined in the present specification).

Abstract: The present invention provides a modified atrial natriuretic peptide that exhibits prolonged duration in blood and maintains cGMP elevating activity. The present invention provides a modified peptide in which at least one sugar substance is linked directly through a glycosidic bond or via a linker structure to at least one hANP peptide, or a pharmaceutically acceptable salt thereof, a medicament comprising the modified peptide or the salt thereof as an active ingredient, etc.

Abstract: It is an object of the present invention to provide a new compound capable of efficiently inducing differentiation from pluripotent stem cells into insulin-producing cells. The object of the present invention is achieved by a compound represented by formula (I): wherein R1, R2, R3, n and A have the same meanings as defined in the description, respectively, or a salt thereof.

Abstract: An object of the present invention is to find a novel pharmaceutical that has an excellent tryptophanase inhibitory effect, and suppresses worsening of renal function to preserve the kidney by reducing production of indoxyl sulfate in the blood. Provided is a pharmaceutical composition containing, as an active ingredient, a compound represented by formula (I) or a pharmacologically acceptable salt thereof. In the formula (I), R1 and R2 are the same or different, and represent a C1-C6 alkyl group, a halogeno C1-C6 alkyl group or a C3-C6 cycloalkyl group; n represents 0, 1 or 2; each X represents a C1-C6 alkyl group, a C3-C6 cycloalkyl group, a halogen atom or the like; and Y represents a hydrogen atom, a C1-C6 alkoxy group, a C3-C6 cycloalkoxy group or a halogeno C1-C6 alkoxy group.

Abstract: The object of the present invention is to improve the post-transplantation engraftment rate of cardiomyocytes that have been purified to such an extent that they are free from non-cardiomyocytes and any components derived from other species. To solve this problem, the present inventors studied the possibility of constructing cell masses from the purified cardiomyocytes. As a result, they revealed that the stated problem could be solved by providing a method of preparing cell masses of cardiomyocytes derived from pluripotent stem cells, characterized in that cell masses of aggregated cells containing cardiomyocytes that had been differentiated and induced from pluripotent stem cells were dispersed to single cells to thereby obtain purified cardiomyocytes, which were then cultured in a culture medium under serum-free conditions so that they were reaggregated.

Abstract: The present invention provides a modified atrial natriuretic peptide that exhibits prolonged duration in blood and maintains cGMP elevating activity. The present invention provides a modified peptide in which at least one sugar substance is linked directly through a glycosidic bond or via a linker structure to at least one hANP peptide, or a pharmaceutically acceptable salt thereof, a medicament comprising the modified peptide or the salt thereof as an active ingredient, etc.

Abstract: The present invention directs to provide a novel compound for treating and/or preventing pain, having excellent skin permeation and reduced skin irritation.

Abstract: The present invention provides a pharmaceutical composition comprising an antibody which binds specifically to human TLR7 or monkey TLR7 and does not bind to mouse TLR7 or rat TLR7, and has an activity of inhibiting a function of human TLR7 or monkey TLR7, and the like.

Abstract: Provided is a novel peptide. The peptide contains the amino acid sequence set forth in SEQ ID NO: 61 and inhibits a protease.

Abstract: It is an object of the present invention to provide an antibody specifically binding to GPR20-positive tumor cells such as GIST, a pharmaceutical product comprising the antibody and having therapeutic effects on a tumor, a method for treating a tumor using the aforementioned pharmaceutical product, and the like. It is another object of the present invention to provide an anti-GPR20 antibody having internalization activity, an antibody-drug conjugate containing the antibody, and the like.

Abstract: The present invention has an object of providing a pharmaceutical drug containing a compound having a good LCAT activation effect on a mutant LCAT protein and a therapeutic effect for LCAT deficiency, and also provides a therapeutic drug for LCAT deficiency containing a compound represented by Formula (I) wherein R1 is a hydrogen atom or a hydroxyl group, and R is a 2-(trifluoromethyl)pyrimidin-5-yl group or a 5-(trifluoromethyl)pyrazin-2-yl group, or a pharmacologically acceptable salt thereof.

Abstract: This application relates to a pharmaceutical composition for use in a method for treating and/or preventing a patient having ectopic ossification and/or brain tumor, wherein the patient has an active mutation in ALK2 protein which is responsible for ectopic ossification or brain tumor; an amino acid residue at position 330 of ALK2 is proline; and an active ingredient of this composition is an anti-ALK2 antibody or an antigen-binding fragment thereof comprising a property of binding to ALK2, a property of cross-linking ALK2, and a property of inhibiting BMP signal transduction.

Abstract: The present invention aims to provide a pharmaceutical drug that contains a compound having an excellent therapeutic effect on inflammatory bowel disease. A therapeutic agent for inflammatory bowel disease, containing as an active ingredient a compound represented by formula (I), wherein R1 represents a hydroxy C1-C6 alkyl group, a C2-C7 alkanoyl group, a C2-C7 alkanoyl C1-C6 alkyl group, a (C1-C6 alkoxy) carbonyl group, a (C1-C6 alkoxy) carbonyl C1-C6 alkyl group, a carboxy group or a carboxy C1-C6 alkyl group, or a pharmacologically acceptable salt thereof.

Abstract: The present invention provides a pharmaceutical composition or a diagnostic composition targeting human fibroblast growth factor receptor 2 (hFGFR2).

Abstract: The present invention establishes a molecular therapy for glycogen storage disease type Ia. The present invention provides an oligonucleotide of 15-30 bases comprising a nucleotide sequence complementary to the cDNA of G6PC gene with c.648G>T mutation, wherein the oligonucleotide comprises a sequence complementary to a region comprising any site between the 82nd to the 92nd nucleotide from the 5? end of exon 5 of the G6PC gene with c.648G>T mutation, a pharmacologically acceptable salt or solvate thereof. Also provided is a pharmaceutical drug comprising the oligonucleotide, a pharmacologically acceptable salt or solvate thereof (e.g., therapeutic drug for glycogen storage disease type Ia).

Abstract: Provided is a short-chain guide RNA that is able to induce site-specific editing even when only a small number of nucleotides is attached to the target recognition site. The guide RNA includes a first oligonucleotide that identifies the target RNA, and a second oligonucleotide that links to the 3? end of the first oligonucleotide. The first oligonucleotide contains: a target-corresponding nucleotide residue that corresponds to an adenosine residue in the target RNA; an oligonucleotide of 15 to 30 residues that links to the 5? end of the target-corresponding nucleotide residue and that has a base sequence complementary to the target RNA; and an oligonucleotide of 3 or 4 residues that links to the 3? end of the target-corresponding nucleotide residue and that has a base sequence complementary to the target RNA. The second oligonucleotide contains 2 to 24 nucleotide residues, and induces site-specific editing of the target RNA.

Abstract: It is an object of the present invention to provide an antibody binding to CDH6 and having internalization activity, an antibody-drug conjugate of the antibody and a drug having antitumor activity, a pharmaceutical product comprising the antibody-drug conjugate and having therapeutic effects on a tumor, a method for treating a tumor using the antibody, the antibody-drug conjugate or the pharmaceutical product, and the like. The present invention provides an anti-CDH6 antibody having internalization activity, an antibody-drug conjugate of the antibody and a drug having antitumor activity, a pharmaceutical product comprising the antibody or the antibody-drug conjugate, and a method for treating a tumor.

Abstract: As an antitumor drug which is excellent in terms of antitumor effect and safety and has an excellent therapeutic effect, there is provided an antibody-drug conjugate in which an antitumor compound represented by the following formula is conjugated to an anti-HER2 antibody via a linker having a structure represented by the following formula: -L1-L2-LP-NH—(CH2)n1-La-(CH2)n2-C(?O)— wherein the anti-HER2 antibody is connected to the terminal L1, and the antitumor compound is connected to the carbonyl group of the —(CH2)n2-C(?O)— moiety with the nitrogen atom of the amino group at position 1 as the connecting position.

Abstract: A method for producing a compound represented by formula (C) wherein R1 represents an amino group protected with a protecting group, the method comprising a step of subjecting a compound represented by formula (B) wherein R1 represents the same meaning as above, to intramolecular cyclization to convert the compound into the compound represented by formula (C).