The purpose of the present invention is to obtain novel therapeutic drugs for malignant tumors, methods for producing a stem cell, or DNA damage-ameliorating agents, etc., wherein a HAT1 and KAT8 inhibitor is used; a kit containing a HAT1 inhibitor and a KAT8 inhibitor may be used; a composition comprising a HAT1 inhibitor, wherein the HAT1 inhibitor is used in combination with a KAT8 inhibitor, may be used; or a composition comprising a KAT8 inhibitor, wherein the KAT8 inhibitor is used in combination with a HAT1 inhibitor, may be used.

The present invention relates to a method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can restore energy failure. More particularly, the invention relates to a method for preventing or treating a cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid.

The present invention provides a method of identifying an agent which can rapidly and accurately regulate activity of androgen receptors in a sebaceous gland- and hair follicle-selective manner. A method of evaluating or selecting an androgen receptor activity regulator selective to sebaceous glands or hair follicles, the method comprising: (A) applying a test agent to cells from sebaceous glands or hair follicles under a hypoxic condition; (B) measuring HIF1 activity in the cells; (C) comparing the HIF1 activity measured in the (B) with HIF1 activity in a control group; and (D) determining a regulatory effect of the test agent on the HIF1 activity based on the result in the (C).

The present invention relates to peptides and compositions for use in improving transduction efficiency of viruses into target cells.

The invention relates to oligonucleotides for inducing skipping of exon 53 of the dystrophin gene. The invention also relates to methods of inducing exon 53 skipping using the oligonucleotides.

Embodiments herein provide engineered mammalian cells, compositions comprising these cells and methods for targeted cancer therapy using cytotoxins derived from Pseudonomas sp. Specifically, it relates to cell-based, targeted in vivo delivery of Pseudonomas exotoxin (PE) for cancer therapy.

Compositions and methods are provided for improved wound healing. In particular, provided herein are compositions and methods for the direct delivery of Sirtuin-1 (Sirt1) or vectors encoding Sirt1 to the wounds (e.g., of diabetic patients). In some embodiments, provided herein are therapeutic devices comprising: (a) a vector encoding Sirtuin-1 (Sirt 1); and (b) a hydrogel carrier. In some embodiments, the vector comprises a viral vector comprising a polynucleotide sequence encoding Sirt 1. In some embodiments, the vector comprises a non-viral vector comprising a polynucleotide sequence encoding Sirt1.

The present disclosure provides methods of treating a human having a disease or disorder that would benefit from increasing platelet counts. The method involves inhibiting the enzyme activity of biliverdin IX reductase (BLVRB) activity or inhibiting the expression of BLVRB gene.

A method for treating an anal or sphincter wound of a subject is described. The method includes administering a therapeutically effective amount of a stromal cell-derived factor-1 (SDF-1) protein or protein variant, or an SDF-1 or SDF-1 variant expression vector in or proximate to the anal or sphincter wound. Topical formulations for administering the SDF-1 or SDF-1 expression vector to an anal or sphincter wound are also described.

The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.