Gene Therapy Research

Your donation is supporting great things

June 17, 2020

Dear friends,

 

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.

 

With love,

The Taouk family

 

An update from the Taouk family

March 02, 2020

Dear friends,

 

We have some great news to share – after searching far and wide and interviewing many talented scientists, CMRI has officially appointed a postdoctoral researcher to join the team as Taouk Family Researcher!

 

They will officially start at CMRI in late March to begin researching PGAP1 mutations and devising a construct for a gene therapy. Although we can’t share their identity just yet, CMRI have told us how excited they are about the skills the researcher will bring to the team with their strong experience in genome analysis, gene screening and molecular biology.

 

Thank you so much again for your support. We have been overwhelmed by your generosity. Your gifts will help cover our researcher’s lab necessities like reagents, models and materials, and potentially help support an additional third year of work for them if we manage to raise enough funding.

 

We are so excited and looking forward to discovering if this research holds the key to a treatment for our girls.

 

The Taouk family

 

Thank you for your support

December 20, 2019

Dear friends,

 

We can’t thank you enough for your support. In 10 days, you have already helped raise nearly $5000 toward the world’s first-ever gene therapy research for PGAP1 mutation.

 

We wanted to say thank you, and to give you a few updates.

 

The University of Sydney has written a beautiful article about our story and what the research will make possible – you can read it here.

 

You might also have seen it shared on the University’s Facebook page. We would so appreciate if you could ‘like’ and share the post so that we can spread the word even further.

 

Our local paper, the Parramatta Advertiser, has also picked up the story – if you are a subscriber, you can read their version here.

 

On behalf of our whole family, we are so grateful for your generosity. We wish you a wonderful holiday season and look forward to updating you on the crowdfunding campaign’s progress in the new year.

 

The Taouk family

 

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