Inhaled mRNA therapy for treatment of cystic fibrosis: Interim results of a randomized, double-blind, placebo-controlled phase 1/2 clinical study.

Background: MRT5005, a codon-optimized CFTR mRNA, delivered by aerosol in lipid nanoparticles, was designed as a genotype-agnostic treatment for CF lung disease.

Methods: This was a randomized, double-blind, placebo-controlled Phase 1/2 study performed in the US. Adults with 2 severe class I and/or II CFTR mutations and baseline ppFEV1 values between 50 and 90% were randomized 3:1 (MRT5005: placebo).

Effects of shape and solute-solvent compatibility on the efficacy of chirality transfer: Nanoshapes in nematics.

Chirality, as a concept, is well understood at most length scales. However, quantitative models predicting the efficacy of the transmission of chirality across length scales are lacking. We propose here a modus operandi for a chiral nanoshape solute in an achiral nematic liquid crystal host showing that that chirality transfer may be understood by unusually simple geometric considerations.

Expression of focal adhesion kinase in small-cell lung carcinoma.

Background: The focal adhesion kinase (FAK) is a non-receptor tyrosine kinase linked to tumor growth, invasion, and metastasis. FAK is overexpressed and associated with prognosis in many cancers, but its prognostic value in small-cell lung carcinoma (SCLC) is unknown and was the focus of this study.

Methods: Total FAK expression was analyzed via immunohistochemistry in tissue microarrays consisting of formalin-fixed, paraffin-embedded SCLC specimens from 85 patients.

Delirium in the nursing home patients seen in the emergency department.

Objectives: To determine whether nursing home patients are more likely than non-nursing home patients to present to the emergency department (ED) with delirium and to explore how variations in their delirium risk factor profiles contribute to this relationship.

Design: Prospective cross-sectional study.

Setting: Tertiary care academic ED.

Errors in drug computations during newborn intensive care.

Medical personnel in a pediatric center were tested for their ability to correctly compute drug doses for sick newborns. One of every 12 doses computed by 95 registered nurses contained an error that would result in the administration of an amount that was ten times higher or lower than the dose ordered. The error rate was no different for experienced or inexperienced nurses.