Use of a mouse-human chimeric anti-α-galactosidase A monoclonal antibody as a reference for measuring serum antidrug antibody titers in patients with Fabry disease.

The detection of antidrug antibodies (ADAs) is important for monitoring patients with Fabry disease who are undergoing enzyme replacement therapy (ERT) with recombinant α-galactosidase A (GLA) drugs, as ADAs can cause allergic reactions and reduce therapeutic efficacy. Various immunological methods, particularly enzyme-linked immunosorbent assay (ELISA), are widely used to measure serum ADA titers in patients with Fabry disease. However, estimating and comparing results of ELISA is challenging because of the absence of a reference antibody.

Peptide-encoding gene transfer to modulate intracellular protein-protein interactions.

Peptide drug discovery has great potential, but the cell membrane is a major obstacle when the target is an intracellular protein-protein interaction (PPI). It is difficult to target PPIs with small molecules; indeed, there are no intervention tools that can target any intracellular PPI. In this study, we developed a platform that enables the introduction of peptides into cells via mRNA-based gene delivery.

Host-directed therapy for bacterial infections -Modulation of the phagolysosome pathway.

Bacterial infections still impose a significant burden on humanity, even though antimicrobial agents have long since been developed. In addition to individual severe infections, the f fatality rate of sepsis remains high, and the threat of antimicrobial-resistant bacteria grows with time, putting us at inferiority. Although tremendous resources have been devoted to the development of antimicrobial agents, we have yet to recover from the lost ground we have been driven into.

Mitochondrial Transfer to Host Cells from Ex Vivo Expanded Donor Hematopoietic Stem Cells.

Mitochondrial dysfunction is observed in various conditions, from metabolic syndromes to mitochondrial diseases. Moreover, mitochondrial DNA (mtDNA) transfer is an emerging mechanism that enables the restoration of mitochondrial function in damaged cells. Hence, developing a technology that facilitates the transfer of mtDNA can be a promising strategy for the treatment of these conditions.

ZLN005 improves the survival of polymicrobial sepsis by increasing the bacterial killing inducing lysosomal acidification and biogenesis in phagocytes.

Polymicrobial sepsis still has a high mortality rate despite the development of antimicrobial agents, elaborate strategies to protect major organs, and the investment of numerous medical resources. Mitochondrial dysfunction, which acts as the center of energy metabolism, is clearly the basis of pathogenesis. Drugs that act on PGC1α, the master regulator of mitochondrial biosynthesis, have shown useful effects in the treatment of sepsis; therefore, we investigated the efficacy of ZLN005, a PGC1α agonist, and found significant improvement in overall survival in an animal model.