I’m a researcher specializing in cystic fibrosis (CF) gene editing and gene therapy. The overall aim of my lab in the Division of Pulmonary Medicine is to develop gene editing strategies that are amenable to clinical translation. My lab was the first to correct a cystic fibrosis mutation using gene editing, and our research is funded by the CF Foundation USA and CF Trust UK.
Following the discovery of CRISPR gene editing in 2012, we’ve published more than 20 papers describing how this Nobel-prize-winning technology can be used to further study and potentially treat CF and other diseases.
I gave a closing keynote talk at the European CF Society conference (June 2023) and will chair the organizing committee of the European CF Society Basic Science Conference (March 2025). I am chair of the CF Trust’s Research Grants Review Committee, have been active in research for over 30 years and began working at Cincinnati Children’s in Spring 2024.
PhD: University of Glasgow, UK, 1992.
BSc: University of Liverpool, UK, 1987.
Pulmonary Medicine
Cystic fibrosis gene editing and gene therapy
Evolution and host-specific adaptation of Pseudomonas aeruginosa. Science. 2024; 385:eadi0908.
Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editing. Molecular Therapy-Methods & Clinical Development. 2023; 31:101140.
Mutation-class dependent signatures outweigh disease-associated processes in cystic fibrosis cells. Cell and Bioscience. 2023; 13:26.
Use of adenine base editing and homology-independent targeted integration strategies to correct the cystic fibrosis causing variant, W1282X. Human Molecular Genetics. 2023; 32:3237-3248.
Cost of gene therapy. Gene Therapy (Basingstoke). 2023; 30:737.
Gene and Rna-Based Therapies. Hodson and Geddes' Cystic Fibrosis. : Taylor & Francis; Taylor & Francis; 2023.
Development of novel therapeutics for all individuals with CF (the future goes on). Journal of Cystic Fibrosis. 2023; 22 Suppl 1:S45-S49.
CFTR RNA- and DNA-based therapies. Current Opinion in Pharmacology. 2022; 65:102247.
CRISPR gene editing - what are the possibilities for respiratory medicine?. Expert Review of Respiratory Medicine. 2022; 16:371-374.
Comparison of Cas9 and Cas12a CRISPR editing methods to correct the W1282X-CFTR mutation. Journal of Cystic Fibrosis. 2022; 21:181-187.