The urgent need for new therapies in TSC
“Research is so important to move us forward, to find new treatments, to stop seizures, to help with kidney manifestations…We’re at a pivotal moment, we have things on the forefront like gene therapy, we have very targeted treatment potential…we can’t stop.”
-Kristen Moore, mother to Jackson with TSC
See more of their story:
What is gene therapy for TSC?
Gene therapies for TSC would deliver a new, functional copy of the TSC1 or TSC2 gene to some cells.
- Treats the genetic “root” cause of TSC, not just symptoms.
- A virus delivers a healthy copy of the TSC1 or TSC2 gene.
- May help many organs, including the brain, lung, and kidneys.
- Aims for lasting, potentially one-time, benefit.
How TSC gene therapy can help families affected
Gene therapy is being developed to deliver a working copy of TSC1 or TSC2 — with the goal of treating the root cause of TSC rather than only managing problems after they appear.
How gene therapy could change care and treatment
While gene therapy is promising because it may one day treat TSC at its source, it still requires more research and time.
TSC1 and TSC2 gene therapy pipeline support
We have a tangible opportunity to change the course of TSC through individualized medicine. Targeting the underlying cause of TSC holds the key for understanding how the disease affects each person so distinctly, which will help inform precise care. Our unique approach to research allows us to engage and leverage innovations across academic and industry stakeholders, fueling progress faster.
How you can help
Through research innovation and partnership, TSC Alliance has determined the path forward in TSC1, having completed initial discovery and testing. In the next 18 months, we have an opportunity to explore new approaches to treating both TSC1 and TSC2, with a laser focus on improved quality of life for everyone impacted. To unlock this next stage of research, we need your help.
Your support fuels breakthroughs in TSC.