Gene Therapy: A New Hope for Spinal Cord Injuries

Gene Therapy: A New Hope for Spinal Cord Injuries

A Groundbreaking Discovery

Scientists at NeuroRestore, Switzerland, have pioneered a gene therapy that stimulates the regeneration of nerve fibres across spinal cord injuries in mice, raising hopes for future applications in humans. The research team discovered that while partial spinal cord injuries often repair themselves, complete injuries do not. This revelation led to the exploration of strategies to enhance the regeneration of nerve fibres, specifically focusing on reconnecting them to their natural targets to restore motor function.

Overcoming Past Challenges

Five years ago, the same team successfully regenerated nerve fibres across complete spinal cord injuries. However, the newly formed fibres failed to connect accurately, preventing the restoration of motor function. In collaboration with UCLA and Harvard Medical School, the team used advanced technology to identify the specific neurons involved in natural spinal cord repair following partial injuries. They found that these axons or nerve fibres needed not only to regenerate, but also to reconnect to their natural targets to restore motor function.

Successful Gene Therapy

Their findings led to the creation of a gene therapy which stimulated the growth of the identified neurons in mice to regenerate their nerve fibres. They also increased specific proteins to support the neuron’s growth through the lesion core and administered molecules to guide the regenerating nerve fibres to their natural targets below the injury. Mice with complete spinal cord injuries were able to walk again, displaying gait patterns similar to those exhibited by mice recovering naturally from partial injuries.

Future Implications and Challenges

This breakthrough has shed light on previously unknown conditions for successful regenerative therapies in restoring motor function after neurotrauma. The scientists predict their gene therapy will complement their existing procedures involving electrical stimulation of the spinal cord. They propose a dual approach treatment for spinal cord injury involving gene therapy for nerve fiber regrowth and spinal stimulation to maximize movement.

While the results are promising, there are still many challenges to overcome before the gene therapy can be tested on humans. However, the team has taken the first steps towards developing the necessary technology. Their findings have been published in the September 22, 2023 issue of Science.