Amelioration of a von Willebrand disease type 2B phenotype in vivo upon treatment with allele-selective siRNAs.

Treatment options for the bleeding disorder von Willebrand disease type 2B (VWD2B) are insufficient and fail to address the negative effects of circulating mutant von Willebrand factor (VWF). The dominant-negative nature of VWD2B makes functionally defective VWF an interesting therapeutic target. Previous in vitro studies have demonstrated the feasibility of allele-selective silencing of mutant VWF using small interfering RNAs (siRNAs) targeting common single nucleotide polymorphisms (SNPs) in the human VWF gene, an approach that can be applied irrespective of the disease-causing VWF mutation.

Spaced Hybrid TiO/Au Nanotube Arrays with Tailored Optical Properties for Surface-Enhanced Raman Scattering.

Controlling the overall geometry of plasmonic materials allows for tailoring their optical response and the effects that can be exploited to enhance the performance of a wide range of devices. This study demonstrates a simple method to control the size and distribution of gold (Au) nanoparticles grown on the surface of spaced titanium dioxide (TiO) nanotubes by varying the deposition time of magnetron sputtering. While shorter depositions led to small and well-separated Au nanoparticles, longer depositions promoted the formation of quasi-continuous layers with small interparticle gaps.

A fully humanized von Willebrand disease type 1 mouse model as unique platform to investigate novel therapeutic options.

Patients suffering from von Willebrand disease (VWD) have reduced quality-of-life despite current treatment options. Moreover, innovation in VWD therapeutic strategies has essentially stalled and available treatments have remained unchanged for decades. Therefore, there is an unmet need to develop new therapeutic strategies for VWD-patients, especially for the large portion of those with VWD-type 1.

Unraveling the Band Structure and Orbital Character of a π-Conjugated 2D Graphdiyne-Based Organometallic Network.

Graphdiyne-based carbon systems generate intriguing layered sp-sp organometallic lattices, characterized by flexible acetylenic groups connecting planar carbon units through metal centers. At their thinnest limit, they can result in 2D organometallic networks exhibiting unique quantum properties and even confining the surface states of the substrate, which is of great importance for fundamental studies. In this work, the on-surface synthesis of a highly crystalline 2D organometallic network grown on Ag(111) is presented.

Exploring the Growth Dynamics of Size-Selected Carbon Atomic Wires with In Situ UV Resonance Raman Spectroscopy.

Short carbon atomic wires, the prototypes of the lacking carbon allotrope carbyne, represent the fundamental 1D system and the first stage in carbon nanostructure growth, which still exhibits many open points regarding their growth and stability. An in situ UV resonance Raman approach is introduced for real-time monitoring of the growth of carbon atomic wires during pulsed laser ablation in liquid without perturbing the synthesis environment. Single-chain species' growth dynamics are tracked, achieving size selectivity by exploiting the peculiar optoelectronic properties of carbon wires and the tunability of synchrotron radiation.