The JAX Rare Disease Translational Center is happy to expand upon its current work with Jack's Tomorrow and PURA syndrome to answer important questions about the safety of potential PURA gene-based therapies.
Exciting news! We’re proud to share that we’re embarking on a new research project with The Jackson Laboratory (JAX) to study the effects of PURA gene overexpression in mice. Gene overexpression studies are vital for advancing gene therapies from the laboratory to clinical trials. They validate genes as safe and appropriate targets for biological therapies and can help guide dosing. We’re hopeful our work with JAX can help guide us towards a potential gene therapy. 🧬 Through this research, we’ll develop a deeper understanding of the PURA gene, as well as key insights for developing future therapies – putting us one step closer to improving the lives of individuals living with PURA Syndrome. Vincent Pons, Ph.D., M.B.A. Associate Study Director at JAX Rare Disease Translational Center, said the new studies “complement our current work on PURA Syndrome and will answer an important question about the safety of potential PURA gene-based therapies.” This work is only possible because of our generous donors. We’re incredibly grateful to be expanding our collaboration with the team of scientists at JAX, who are on the cutting-edge of rare disease research. Learn more: https://lnkd.in/eC9_2DTj