We hope you are all well, and keeping safe in these challenging and unprecedented times.
As you can imagine, Mary and Neveah are particularly susceptible to viruses like COVID-19 due to their weakened immune systems, so we are keeping them safe at home as much as we can. We’re also thinking about the many others out there, young and old, who are similarly vulnerable. At times like these we are even more grateful that medical researchers are working hard to make the world a safer and healthier place.
We also wanted to give you an update on how the Taouk Family Postdoctoral Researcher is going. At almost three months into the role, we’ve been told they’ve hit the ground running with this research, with the help of your generous donations.
The researcher is halfway through designing the first draft gene therapy construct for PGAP1. Many drafts will need to be developed and tested, so we’re very excited that this important first step is already underway. They have also organised for sample PGAP1 cells to be reprogrammed into pluripotent stem cells, which will form the basis of many models that will be used to test the individualised gene therapeutics developed as part of this program.
These pluripotent stem cells are like ‘master’ cells that can basically be directed to develop into nearly any other type of cell in the body – such as liver cells, heart cells, or in this case neuron cells. By growing PGAP1-affected neuron cells in a dish or another model, it means that scientists can see how their therapy might perform ‘in real life’ in a patient’s brain – resulting in greater accuracy for the final therapeutic solution.
If you’d like to learn more about how gene therapy works, Dr Leszek Lisowski (who shares supervision of our researcher) from the University of Sydney and Children’s Medical Research Institute explains the topic in these podcasts:
- Interview with James Valentine on the ABC: click here to view the full transcript.
- ‘Wide Open Air Exchange’ podcast – click here to listen to Dr Lisowski’s explanation of vectorology and genome editing and how viral vectors are used in gene therapy.
We know that everyone is doing it tough right now, and we’re incredibly grateful for all the help you have given us so far. If you or anyone you know might like to support this research, please let them know they can still donate via the crowdfunding page. All donations will help fast-track gene therapy research into PGAP1 undertaken by our researcher and are 100% tax-deductible.
Thank you so much again, everyone, and please stay safe.
The Taouk family