Motor neuron disease (MND) Research

ATXN2 gene therapy


Testing a novel drug delivery technique to deliver ATXN2 gene therapy

Dr Andrea Perera, Professor Chris Shaw, Professor Keyoumars Ashkan & Dr Olivier Brock, King’s College London

Up to £250,000 | March 2023 - February 2025

Summary: In around 97% of people living with MND, a protein called TDP-43 accumulates excessively in motor neurons, leading to their degeneration. 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. 

To find out more about TDP-43, check out our infographic.

To find out more about novel drug delivery techniques, check out our infographic.

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