. Adenine base editing with engineered virus-like particles rescues the CFTR mutation G542X in patient-derived intestinal organoids. iScience. 2025; 28:111979.
. Prime editing: therapeutic advances and mechanistic insights. Gene Therapy (Basingstoke). 2025; 32:83-92.
. 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.
. In Vitro and In Vivo Models to Study Nephropathic Cystinosis. Cells. 2022; 11:6.
. Spotlight on gene therapy in Germany. Gene Therapy (Basingstoke). 2021; 28:471-472.
. Prime editing - an update on the field. Gene Therapy (Basingstoke). 2021; 28:396-401.
. CRISPR Deletion of a SVA Retrotransposon Demonstrates Function as a cis-Regulatory Element at the TRPV1/TRPV3 Intergenic Region. International Journal of Molecular Sciences. 2021; 22:1911.