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Rachel Ashworth

BSc (Hons) Biochemistry


Research Assistant

My research focuses on gene therapy for cystic fibrosis. Cystic fibrosis is a genetic disease, resulting from a mutation in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, encoding the CFTR ion channel. Cystic fibrosis is characterised by abnormal epithelial transport in the apical membrane of secretory cells, effecting epithelial mucus secretions. Mortality is usually related to the thick mucus build up in the lungs leading to respiratory infections and lung damage. 

Improvements in Cystic Fibrosis care have greatly improved the quality of life and life expectancy of cystic fibrosis patients; however, most therapies treat the symptoms not the underlying cause of the disease. Only one therapy corrects the underlying cause of the disease but this is only available to 4-5% of patients who have the G551D mutation. We are working on a lentiviral vector to deliver the correct version of the CFTR gene to the lungs, this approach is suitable for all cystic fibrosis sufferers irrespective of the underlying mutation. I am working on transferring our lead lentiviral vector candidate to a CMO, for large scale production prior to the initiation of the first-in-human clinical trial. My research involves optimisation of the lentiviral vector production process along with assay development and optimisation, particularly TaqMan Real Time PCR and RT-PCR, as well as pre-clinical studies to assess vector efficacy.  

I have a first class BSc (Hons) in Biochemistry from the University of Birmingham. Prior to joining the Gene Medicine group in October 2016 I worked for a leading gene and cell therapy company in process research and development.

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