Phase 1/2 trial of brogidirsen: Dual-targeting antisense oligonucleotides for exon 44 skipping in Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD) is a severe muscle disorder caused by mutations in the DMD gene, leading to dystrophin deficiency. Antisense oligonucleotide (ASO)-mediated exon skipping offers potential by partially restoring dystrophin, though current therapies remain mutation specific with limited efficacy. To overcome those limitations, we developed brogidirsen, a dual-targeting ASO composed of two directly connected 12-mer sequences targeting exon 44 using phosphorodiamidate morpholino oligomers.

Novel approach for quality control testing of medical displays using deep learning technology.

In digital image diagnosis using medical displays, it is crucial to rigorously manage display devices to ensure appropriate image quality and diagnostic safety. The aim of this study was to develop a model for the efficient quality control (QC) of medical displays, specifically addressing the measurement items of contrast response and maximum luminance as part of constancy testing, and to evaluate its performance. In addition, the study focused on whether these tasks could be addressed using a multitasking strategy.

Development and validation of an algorithm for identifying patients undergoing dialysis from patients with advanced chronic kidney disease.

Background: Identifying patients on dialysis among those with an estimated glomerular filtration rate (eGFR) < 15 mL/min/1.73 m remains challenging. To facilitate clinical research in advanced chronic kidney disease (CKD) using electronic health records, we aimed to develop algorithms to identify dialysis patients using laboratory data obtained in routine practice.

Pseudotunnel Magnetoresistance in Twisted van der Waals FeGeTe Homojunctions.

Twistronics, a novel engineering approach involving the alignment of van der Waals (vdW) integrated two-dimensional materials at specific angles, has recently attracted significant attention. Novel nontrivial phenomena have been demonstrated in twisted vdW junctions (the so-called magic angle), such as unconventional superconductivity, topological phases, and magnetism. However, there have been only few reports on integrated vdW layers with large twist angles θ, such as twisted interfacial Josephson junctions using high-temperature superconductors.

Bone marrow niches orchestrate stem-cell hierarchy and immune tolerance.

Stem cells reside in specialized microenvironments, termed niches, at several different locations in tissues. The differential functions of heterogeneous stem cells and niches are important given the increasing clinical applications of stem-cell transplantation and immunotherapy. Whether hierarchical structures among stem cells at distinct niches exist and further control aspects of immune tolerance is unknown.