Neurology Central

SfN19: Gene therapy advancements may aid future treatments for neurodegenerative diseases

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Abstracts highlighting the potential of gene therapy in the treatment of a range of neurodegenerative diseases were presented at SfN Neuroscience (19–23 October 2019, Chicago, IL, USA). Studies in mouse models and in human brain tissue demonstrated promising results.

“The research presented today represents important and exciting steps toward being able to prevent and treat disorders that currently have no cure, such as Parkinson’s disease and Alzheimer’s disease,” commented Jeff Kordower, a Professor at Rush University (IL, USA).


Gene therapy approach reprograms reactive astrocytes into motor neurons to restore motor function in a mouse model of amyotrophic lateral sclerosis

One study that was presented aimed to use in vivo cell conversion to tackle the loss of motor function associated with amyotrophic lateral sclerosis (ALS). The research group, from Penn State University (PA, USA), utilized adeno-associated virus (AAV)-based gene therapy to reprogram astrocytes into motor neurons in an ALS mouse model.

They observed an increased number of motor neurons in the spinal cords of the treated mice. In addition, the group reported a significant improvement in motor function of the animals and an extended lifespan.

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