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Gene Therapy Injection into Spinal Cord Halts ALS in Adult Mice
Published:06 Dec.2018    Source:News Scientist

A new delivery method could take us a step closer to a gene therapy for amyotrophic lateral sclerosis (ALS), a neurodegenerative disorder in which nerve cells progressively stop working throughout the spinal cord and the brain.


Animal studies have already suggested that ALS can be prevented by replacing the mutated genes that cause some forms of the condition with normal versions. But delivering genes to nerve cells in the spine is a challenge.The new method involves injecting corrected genes beneath the tissues that protect the spinal cord. The technique has been shown to correct 89 per cent of genes associated with an inherited form of ALS.