Cystic fibrosis (CF) is the most common life-threatening recessive genetic condition affecting Australian children. While there is no cure, the faulty gene responsible for the condition has been identified as the CFTR gene, and research now focuses on how to repair or replace it.
Harsha Padmanabhan’s research aims to contribute to this goal by developing an aerosol spray for delivering the lentiviral vector carrying the correct CFTR gene.
Gene therapy involves inserting a working copy of the faulty gene. The concept is to use a viral vector (lentivirus) to carry the CFTR copy, and insert it into the CF patient’s lung. Harsha’s research is looking at developing this gene therapy into an aerosol spray, which will enable it to be delivered non-invasively.
This would be an incredibly useful application, as we head towards clinical trials of gene therapy for CF in the future.
“I’ve always wanted to do cutting edge research in the field of genetic medicine,” says Harsha. “The molecular concepts behind gene therapy and gene delivery are very interesting, and the fact that I could make a difference in someone’s life gives me the inspiration to pursue this research.”
With a Bachelor of Science in Biotechnology from Manipal University in India and a Master of Human Biotechnology from Flinders University, Harsha joined the University of Adelaide as a research assistant for Bioconst Pty Ltd at the Waite campus, specialising in plant tissue culture.
In late 2012, she joined the Adelaide Cystic Fibrosis Gene Therapy Research Group in the University’s Robinson Research Institute as a PhD student.
Harsha is a recipient of the Women and Children’s Hospital MS McLeod Scholarship, Cystic Fibrosis Australia’s Top-Up Scholarship to help support her studies and a European Molecular Biology Laboratory scholarship for a training course conducted in Melbourne. In 2014 she won the 3MT competition at the University of Adelaide and went on to represent the University at the Trans-Tasman Three Minute Thesis competition held in Perth.