miRNA markers decreased in extracellular vesicles (EVs) in the blood of ALS patients and therapeutic drugs that replenish these miRNAs
Discovery of mGluR5 dysfunction in ALS patients with TDP-43 pathology offers a new direction for the development of disease-modifying therapies
This antisense oligonucleotide (ASO) sterically blocks the binding hotspots of RNA-binding proteins to reactivate the splicing of an "exitron" within the TDP-43 gene. As a result, it reduces the production of the Intrinsically Disordered Region (IDR) that triggers aggregation, while simultaneously increasing a protective short isoform (ΔIDR). This isoform guides TDP-43 aggregates to be processed via a "Cap & Escort" mechanism. This approach simultaneously achieves the typically conflicting goals of suppressing cytoplasmic aggregation and restoring nuclear function.
Upstream Stop Codon Insertion to Suppress CAG Repeat–Driven Disease Progression
Development of Molecular Targeted and Nucleic Acid Therapeutics for C11orf97
Target molecules and their inhibitors that control the survival and reactivation of disseminated tumor cells (cancer metastasis-initiating cells).
Inserting 50-nucleotide synthetic repeat sequences into the 5’UTR significantly enhances mRNA translation, achieving up to 10-fold increases in protein expression in both cells and animals
Discovery of a new mechanism of hypertension induction and proposal for drug discovery collaboration based on the mechanism.
New therapeutic targets and therapeutic agents for trauma-induced heterotopic ossification in FOP patients.
D35LNPs stimulate TLR9 and significantly induce the release of IFN-α from plasmacytoid dendritic cells, thereby strongly enhancing systemic immunity
siRNAs containing innovative artificial nucleic acids selectively suppress mRNAs containing abnormally expanded CAG repeats
Improved S-TuD (Synthetic Tough Decoy) RNA nucleic acid that inhibits miR-200 family, which is highly expressed in mammary cancer, colon cancer, endometrial cancer, ovarian cancer, etc.
Using vesicles derived from the cell membrane of target bacteria allows for highly species-specific treatment
Identification of prostaglandin receptors involved in the pathogenesis of endometriosis. [Collaborative Research Proposal]
Therapeutic target molecules and antisense oligonucleic acid (ASO) therapeutic agents for autosomal dominant polycystic kidney disease (ADPKD).
Splice-switching antisense oligonucleotides (SSOs) targeting the α-synuclein (SNCA) gene suppress α-synuclein (α-Syn) aggregation.
ASO-4733: Optimized Anti-SYT13 antisense oligonucleotides
Newly designed KK-(EK)4-lipid having near neutral charge enhances intracellular translocation of drug carrier
Molecular targeted drugs targeting the NIK associated with the differentiation factor from hepatocytes to cholangiocarcinoma
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