Paul B. McCray

Optimized AAV capsids robustly transduce airway epithelial cells

This paper develops novel AAV capsids with improved transduction efficiency for airway epithelial cells, reducing required doses and enhancing therapeutic potential for respiratory diseases.

Novel mouse reporter models for the detection of genome editing events in vivo

The paper develops new mouse reporter models to detect genome editing events in vivo, including gene repair and CRISPR indels, and A-base editor activity.

Lentiviral-mediated gene complementation rescues pathogenic ABCA3 variants

This study tests lentiviral-mediated ABCA3 gene complementation in cell lines with specific pathogenic variants, showing partial rescue of cellular functions and differential effects based on variant type.

In Utero CFTR Modulation Alleviates Disease in G551D Cystic Fibrosis Pigs

This paper shows that in utero ivacaftor treatment rescues multiple organ defects in CFTR-G551D pigs, improving pancreatic, gallbladder, and vas deferens function.

IL-13 decreases susceptibility to airway epithelial SARS-CoV-2 infection but increases disease severity in vivo

This study shows IL-13 protects airway epithelial cells from SARS-CoV-2 infection in vitro but worsens disease severity in mice through eicosanoid signaling.

A predominately pulmonary activation of complement in a mouse model of severe COVID-19

This study identifies pulmonary complement activation in a mouse model of severe COVID-19, showing local complement production and its role in lung injury.

High ionic strength vector formulations enhance gene transfer to airway epithelia

Development and Initial Characterization of Pigs with DNAI1 Mutations and Primary Ciliary Dyskinesia

This paper develops a PCD pig model with DNAI1 mutations and characterizes its respiratory and sinus disease features, showing similarities to human PCD.

Lentiviral vectors transduce lung stem cells without disrupting plasticity

This study shows that lentiviral vector transduction of airway basal cells does not impair their differentiation or function, suggesting potential for long-term gene therapy in lung diseases.

Engineered transfer RNAs for suppression of premature termination codons

This paper identifies engineered transfer RNAs (ACE-tRNA) that suppress premature termination codons in mammalian cells and mice, showing high suppression activity for common disease-causing nonsense codons.