ATXN2 gene therapy
Professor Chris Shaw, Dr Andrea Perera, Professor Keyoumars Ashkan and Dr Olivier Brock, King’s College London
Advancing Treatments Award
Accelerate new treatments
March 2023 - May 2026
Up to £250,000
Summary
TDP-43 is a protein that plays an important role in the assembly line that converts information stored in our DNA into molecules called RNA, which are used as the template for making the proteins within our cells. TDP-43 is known to be dysregulated in about 97% of people living with MND and forms toxic clumps in motor neurons. This process has been linked to the activity of another protein called ataxin-2. Dr Perera and her team are developing a gene therapy that targets ataxin-2, to reduce TDP-43 build-up, thereby addressing a key cause of MND.
This project uses an innovative new drug delivery method, called subpial injection, which administers gene therapy directly beneath the brain and spinal cord's protective layer, enhancing delivery to motor neurons and treatment effectiveness.
Why this research is important
If successful, this research could pave the way for clinical trials in humans, potentially offering a new treatment option for people living with MND. Moreover, the subpial injection technique could have broader applications, enhancing the delivery of various drugs to the brain and spinal cord, thereby advancing the field of drug delivery for neurological disorders.
Read here to learn more about this project. Learn more about TDP-43, gene therapies and drug delivery techniques through our linked infographics.
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