Matthew David Disney

This study develops a synthetic cleavage module that selectively degrades pathogenic r(CUG) repeats in cells, reducing DM1-associated pathology without DNA damage.

SMARTFlexDB links ligand-bound and ligand-free RNA structures to study conformational flexibility and small-molecule recognition, providing a resource for RNA-targeted drug discovery.

This paper develops a programmable platform to recruit RNA-binding proteins using small molecules, enabling RNA destabilization, and demonstrates its application to MAPT pre-mRNA.

SMARTPocket uses geometric deep learning to predict RNA-small molecule binding pockets from 3D structures, outperforming existing methods and identifying cryptic ligandable sites.

This study shows that degrading the miR-21 precursor with TGP-21-RiboTAC reduces pathogenic pathways in fibrosis models by reactivating tumor suppressor genes.

The paper develops a chemoinformatics platform to discover RNA-binding small molecules, identifying ligands for the r(CUG)exp RNA in DM1 with splicing rescue activity.

The paper studies how dynamic conformational changes in pre-miR-377's Dicer site enable small-molecule recognition, showing that binding depends on RNA dynamics rather than a single structure.

This paper introduces epoxide- and aziridine-2-carboxamide electrophiles that covalently target RNA with stereoselectivity, demonstrating their application to RNA targets like r(CUG)exp repeats and FMN riboswitches.

The paper identifies bioactive ligands for the toxic r(G4C2)exp RNA using DNA-encoded libraries combined with docking and SAR analysis.

The paper develops a live-cell method to identify RNA-small molecule interactions and uses it to design selective RNA degraders.

This study designs a covalent small molecule targeting unpaired guanines in a riboswitch, showing it modulates riboswitch function in a cellular reporter system.

SMARTBind uses deep learning and contrastive learning to identify small molecule binders to RNA without structural input, outperforming existing methods and reducing computational cost.

The paper identifies electrophilic compounds that covalently bind RNA and uses this insight to design an RNA-specific covalent binder for myotonic dystrophy type 1.

This paper designs an orally bioavailable small molecule that targets RNA to reduce 4R tau protein abundance and alleviate disease phenotypes in a mouse model.

This paper describes a method to map small-molecule RNA-binding sites in live cells and identifies their targets and binding sites.

This paper reports NMR structures and magnetic force spectroscopy studies of small molecules binding to RNA CAG repeat expansions, revealing their binding modes and structural effects.

The paper reports NMR structures of small molecules bound to an RNA CUG repeat expansion motif, revealing binding interactions and conformational changes that inform drug design.

This study develops isogenic iPSC lines with MAPT S305 mutations to increase 4R tau expression, revealing altered neuronal and astrocytic functions in tauopathies.

This paper uses ScanFold to map the RNA structural landscape of SARS-CoV-2 and identifies potential drug targets and antisense therapeutic regions.

This paper shows that toxic RNA repeats can catalyze the synthesis of their own inhibitors, leading to degradation and rescue of DM2-related defects.

The paper identifies structured regions in tau pre-mRNA that influence splicing and stability, potentially offering new therapeutic targets for tauopathies.

The paper identifies stable secondary structures in MYC mRNA, including a conserved 3′ UTR motif that affects gene expression and offers potential druggable targets.