New Genes® was founded to fund desperately needed research in the cutting edge arena of Gene Replacement Therapy. Perhaps not surprisingly, we founded New Genes® as our family has been personally touched by the devastation that is wreaked by a set of bad genes. In October 2016 our beautiful then 6 year old was diagnosed with Juvenile Batten Disease (CLN3) – a rare, neuro-degenerative childhood disease caused by inheriting a recessive, defective gene from Mum and Dad each. Our little boys only hope lies in giving him new genes via Gene Replacement Therapy.
New Genes® exists to fund research into Gene Replacement Therapy as a whole, and the research is not isolated to CLN3. It is our firm belief that this amazing and emerging area of research will see the demise of many diseases that devastate the lives of families and individuals.
Our vision is to bring hope to the hundreds of thousands of human beings who suffer from a disease due to a defect in their DNA. Gene Replacement Therapy has the potential to slow or even halt the progression of many diseases that affect our community including thousands of innocent children.
WHAT IS GENE REPLACEMENT THERAPY?
Essentially Gene replacement therapy is a genetic technique that allows for the insertion of a ‘working’ copy of the defective gene that is causing the specific disease.
A modified virus or vector is used to deliver the corrected gene into the body. For example the AAV9 vector is used to deliver CLN3 into mice. The Lentivirus vector is used to deliver the PGC1-alpha into mice to prevent Alzheimer’s disease.
Imagine doctors having the ability to treat a disorder by inserting a gene into a patient's cells instead of using drugs or surgery. The possibilities are endless once the crucial task of establishing how best to ‘introduce’ the working copy of the gene into the area of the body that requires it can be met.
What is Juvenile Batten Disease?
Juvenile Batten Disease (CLN3) is a rare, fatal, inherited disorder of the nervous system that typically begins in childhood. The first symptom is usually progressive vision loss in previously healthy children followed by personality changes, behavioural problems and slow learning. Seizures commonly appear within 2-4 years of vision loss. However, seizures and psychosis can appear at any time during the course of disease. Progressive loss of motor functions (movement and speech) start with clumsiness, stumbling and Parkinson-like symptoms; eventually, those affected become wheelchair-bound, are bedridden, and die prematurely.
HELP US GET TO HUMAN TRIAL STAGE!
Clinical trials in animal models have showed promising results for the treatment of Juvenile Batten Disease via Gene Replacement Therapy. The next phase is human clinical trials. This is an exciting yet costly phase for any research project. AND IT IS WHERE WE NEED YOUR HELP. We are SO close to saving the young lives of the beautiful children affected by Juvenile Batten Disease. And once we beat Batten Disease, our foundation will fund research into another horrible genetic childhood disease. Please DONATE NOW.