Gene Therapy Injection Into Spinal Cord Halts ALS in Adult Mice

A new gene therapy delivery method involving injecting corrected genes beneath the tissues that protect the spinal cord has been shown to correct 89 per cent of genes associated with an inherited form of ALS.

Amyotrophic lateral sclerosis (ALS), is a neurodegenerative disorder in which nerve cells progressively stop working throughout the spinal cord and the brain.

Animal studies have shown that by replacing mutated genes that cause some forms of the condition with normal versions, ALS can be prevented. The challenge has been delivering genes to nerve cells in the spine.

There is currently no effective treatment for this familial form of ALS or the more common sporadic ALS, both of which have a median survival of 3 to 5 years after the onset of symptoms.

If the trials continue to go well and continue to a therapy for humans, people may begin to use genetic screening to find out if their family is genetically at risk for this form of ALS, and treating the disease before symptoms begin.



Image: “Mouse spinal cord neuron2” by NICHD is licensed under CC BY 2.0