Progress and prospects in antisense oligonucleotide-mediated exon skipping therapies for Duchenne muscular dystrophy.

Recent years have seen enormous progress in the field of advanced therapeutics for the progressive muscle wasting disease Duchenne muscular dystrophy (DMD). In particular, four antisense oligonucleotide (ASO) therapies targeting various DMD-causing mutations have achieved FDA approval, marking major milestones in the treatment of this disease. These compounds are designed to induce alternative splicing events that restore the translation reading frame of the dystrophin gene, leading to the generation of internally-deleted, but mostly functional, pseudodystrophin proteins with the potential to compensate for the genetic loss of dystrophin.

AP39, a novel mitochondria-targeted hydrogen sulfide donor, promotes cutaneous wound healing in an in vivo murine model of acute frostbite injury.

Frostbite injury refers to cold tissue injury which typically affects the peripheral areas of the body, and is associated with limb loss and high rates of morbidity. Historically, treatment options have been limited to supportive care, leading to suboptimal outcomes for affected patients. The pathophysiology of frostbite injury has been understood in recent years to share similarity with that of cold ischemia-reperfusion injury as seen in solid organ transplantation, of which mitochondria play an important contributing role.

Assessing the potential acoustic impact of floating offshore wind farms in the Central Mediterranean Sea.

The Strait of Sicily, a vital marine passage with diverse fauna, is seeing a steep rise in the planning of offshore wind farm projects. This study assesses the acoustic impact of these wind farms on local marine species. Underwater propagation was modeled for three proposed floating wind farms using JASCO's Marine Operations Noise Model (MONM), which integrates a parabolic equation method for frequencies from 10 to 800 Hz and a beam-tracing model for 1 to 25 kHz.

Tissue-specific modulation of CRISPR activity by miRNA-sensing guide RNAs.

Nucleic acid nanostructures offer unique opportunities for biomedical applications due to their sequence-programmable structures and functions, which enable the design of complex responses to molecular cues. Control of the biological activity of therapeutic cargoes based on endogenous molecular signatures holds the potential to overcome major hurdles in translational research: cell specificity and off-target effects. Endogenous microRNAs (miRNAs) can be used to profile cell type and cell state, and are ideal inputs for RNA nanodevices.

Nitrogen Enriched Tröger's Base Polymers of Intrinsic Microporosity for Heterogeneous Catalysis.

Heterogeneous catalysis is significantly enhanced by the use of highly porous polymers with specific functionalities, such as basic groups, which accelerate reaction rates. Polymers of intrinsic microporosity (PIMs) provide a unique platform for catalytic reactions owing to their high surface areas and customizable pore structures. We herein report a series of Tröger's base polymers (TB-PIMs) with enhanced basicity, achieved through the incorporation of nitrogen-containing groups into their repeat units, such as triazine and triphenylamine.