Taking CF research to the Houses of Parliament
I studied my Biology degree and Immunology Master’s degree at the University of Granada, Spain, before I moved to Newcastle University. I have always been interested in rare diseases and how scientific research can help find better treatments.
I was delighted when I was selected for this PhD position to develop new models of normal and cystic fibrosis (CF) airway cells for CF drug discovery, using gene editing of human stem cells. These cellular models are cells that have been grown in the lab and have been edited to contain a CF-causing mutation, and are required for much of the research undertaken by scientists in the CF field. This PhD forms part of a Cystic Fibrosis Trust-funded Strategic Research Centre (SRC) INOVCF, a multidisciplinary international project involving researchers from Lisbon, Regensburg and Chapel Hill in the US, looking at new ways to treat cystic fibrosis.
Tackling the big issues in CF research
Under the supervision of Dr Mike Gray and Professor Neil Perkins, I am trying to overcome a very important issue in the CF drug discovery process, which is the low availability of good in vitro (meaning for lab-based research) human cellular models that can be used for discovering new treatments for cystic fibrosis. I am taking advantage of revolutionary gene-editing technologies to introduce the ΔF508 mutation, the most frequent mutation that causes CF, into stem cells which can then, using a specialized protocol, be pushed to become lung epithelial cells for future drug discovery. So far, I have succeeded in gene editing the healthy human stem cells to produce CF stem cells, and the next step is to direct these CF cells into becoming the functional lung cells that we wish to study.
With this project I hope to help speed up the CF drug discovery process and, therefore, to contribute to finding more effective treatments for people with cystic fibrosis.
An exciting opportunity
I am very pleased that I have been chosen to present my research at the STEM for Britain event. This is an excellent opportunity not only to make MPs, and the public in general, more aware of CF, but it will also bring important issues closer to people who ultimately can influence the course of our society. I am also really looking forward to finding out about the best research that is going on in the UK. I really hope to get the message across that "good research is an essential part of our future development. It all starts with a good project proposal supported by generous funders and it continues with the effort of many scientists working together for a common goal."
In addition to the Cystic Fibrosis Trust and its supporters, I am also very grateful to Disease Models & Mechanisms for funding my travel to Professor Janet Rossant’s lab in Toronto, Canada, a collaborator on our INOVCF project, where I learnt how to produce lung cells from stem cells.
A glowing review
Sara's supervisor, Dr Mike Gray, is the principal investigator on the Trust's INOVCF SRC. He said: "I am very proud that Sara was chosen to present her work at the STEM for Britain event. Sara is a highly dedicated young scientist and I am sure she will find this an inspirational event. It is a great opportunity to talk to people of national influence about her exciting research, which we hope will ultimately help people with cystic fibrosis."
If you're inspired by Sara's work, you can make a donation and help us continue to invest in groundbreaking research into cystic fibrosis.