FoxG1

TOGETHER WE WILL FIND A CURE

Imagine if you were not able to sit, Imagine if you are not able to walk, Imagine if you were not able to talk by yourselves, these are some of the challenges faced by children affected by FoxG1 syndrome everyday! Now imagine if we could change all that. What IF you could make a difference? The goal of FoxG1 Foundation Australia is to provide hope and support for individuals with FOXG1 and their families via any means possible. We are their voice.

Daniel at UNSW

Research

Phenomenal advances in genetics and research are enabling ground breaking discoveries everyday.

CRISPR  the new gene editing technique that sparked intense interest since 2014 as made gene editing easier cheaper faster, and more efficent than ever before.

As FoxG1 is a monogeneic disorder, the hope of fixing the errenous mutation is very high using CRISPR

Sometimes even awareness of rare diseases is a challange, with FoxG1, research has started at UNSW which is  a great start. This sis a long journey and the first steps have been taken . Daniel Tan is the researcher working on FoxG1 under Fabiens guidance

Gene edit

Genetics

The human body has 20,000 genes. Genes are like little machines that make protiens. FoxG1 is one of those genes that is responsible for development of the brain.

While mutations in genes are more common than is percived , resulting in minor changes like differences in colour of our eyes or hair, sometimes errors or mutations on critical genes like the FoxG1 gene result in profoundly debilitating conditions.

The error on genes is like one mising link in a chain tha is say 2 kiloneters long.

With phenomenal advances in computing and nanotechnology, gene sequencing or checking has become very affordable and so the causes of many previously undiagnosable conditions is becoming more apparent. As an example Kushara was the first child in Sydney ever to be diagnosed with FoxG1 syndrome and No5 in Australia.

 

 

Layla_pic

Hope

2014 saw the worlds first case of gene editing resulting in saving the life of a girl and Lyla made headlines around the world.

2015 and 2016 also saw the commerciliaziation of Glybera and Strimvelis. These are gene therapy cures that have been commercialized and are available now. These drugs were built on older gene editing techniques calles TALEN's.

The discovery and adoption of CRISP is bringing in the dawn of a new era of genetic cures. These along with phenomenal advances in computaional power are leading to great leaps in understanding of geneic diseases and their treatment therapies. There are over 200 gene therapy clinical trials underway. We are in the middle of a genetic revolution.

All of these gene therapies and the associated research in getting them there is building a massive amount of learning, expertise and capability around making these cures possible.

On Valentine 's day in 2017, the NIH/NIM gave approval for gene therapy in principal. This was a day more significant than the trinity test as this marks the dawn of an unprecedent era of change in healthcare.

TOGETHER WE WILL FIND A CURE FOR FOXG1 TOO

Together we can end childhood suffering