Children’s Medical Research Institute (CMRI) is excited to announce the founding of a new Postdoctoral Researcher position, focused on cystic fibrosis (CF), thanks to the generosity of the Team Simon Foundation which has a very personal connection to the cause.

Team Simon Foundation is the brainchild of Teresa and Harry Bazouni who want to see treatments and cures developed for kids with CF, a condition affecting their son Simon and many thousands of children worldwide.

In May 2014, Simon was born seemingly healthy before things rapidly changed. Over the following months, Teresa and Harry watched him struggle to breathe before he was finally diagnosed with CF, which impacts the lungs……….. and the whole family’s life changed forever.

“It was a big shock – a devastating day,’’ Teresa said. “It was overwhelming.’’

The daily routine for a child with CF involves physiotherapy, using an inhaler to clear out their chest, and 25 pills a day.

“A lot of people don’t know what a genetic disease is,’’ Harry said. “Simon’s condition is invisible. From the outside he looks all nice and healthy, but everything’s happening on the inside.’’

His family was determined to change the future for Simon and other children with CF, so they founded Team Simon Foundation. Connecting with CMRI, through Jeans for Genes™, the family learnt about the gene therapy revolution.

Instead of daily treatments for genetic diseases, gene therapy aims to treat, or even cure, a patient through one single therapeutic administration.

Head of CMRI’s Translational Vectorology Unit, Associate Professor Leszek Lisowski, and his team have been researching ways to use gene therapy to treat CF.

Associate Professor Lisowski’s team creates microscopic delivery vehicles, called vectors, which make gene therapies possible. The vectors deliver gene therapeutics into the patient’s cells affected by the disease. The therapy adds a working copy of the faulty gene or corrects the faulty gene with a functional version.

“Cystic fibrosis impacts the lungs, so in order for us to deliver a gene therapy, we have to go directly into the lungs. This is especially difficult in the case of CF, where the airways are covered with a thick mucus that is impenetrable to therapeutic vectors.’’ Associate Professor Lisowski said.

“We are thus working to develop novel bioengineered viral vectors that would enable safe and effective delivery of gene therapeutics to the affected lung cells via the blood stream. If successful, this new technology could be life-changing for patients with cystic fibrosis but also other lung disorders.”

“As an independent medical research institute, we are not guaranteed government funding so we are extremely grateful to the Team Simon Foundation for investing in our work, which we hope will benefit current patients as well as future generations of children.’’

The Team Simon Foundation’s pledge of $464,000 will fully fund the new postdoctoral position over three years.

Teresa and Harry said they wanted to create a legacy for others in their situation.

“We have toured the labs, we have met the researchers, and we have great faith in the potential for them to make a real difference in the lives of children living with CF.’’

If you would like to assist the family to reach their pledge goal, visit: https://myfundraiser.cmri.org....