Overcoming the Complexities With Gene Therapy in ALS: Anna Underhill, BS – Neurology Live

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For approximately 90% of all cases of amyotrophic lateral sclerosis (ALS), there is no known family history of the disease or presence of a genetic mutation linked to the disease. Although, for the remaining 5% to 10%, there is a known family history of the disease that has opened the door for gene therapy, a targeted approach that can potentially fix or block the negative effects of coding errors. Some of the most promising gene-therapybased approaches for ALS to date include antisense oligonucleotides, RNA interference, or gene editing technology such as CRISPR.

With close to 40 different genes discovered that are linked to the disease, the main issue is now validating the genes. One of these, SARM1, a variant thought to promote neuron death, is also being evaluated by several researchers from Kings College London. The project will use stem cells converted to neurons in a laboratory to firmly establish a causative role for the variant, as well as manipulate the cell culture environment and other genes to identify risk factors interacting with it.

There are several reasons why the research has not translated to therapeutic success, says Anna Underhill, BS, an investigator of the project. Underhill, a postdoctoral researcher at Kings College London, sat down with NeurologyLive to discuss the complexities of gene therapy, where the lag stems from, and the future directions of her study. She also provided detail on the uniqueness of SARM1 mutation, including a possible compensatory mechanism in the cell.

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Overcoming the Complexities With Gene Therapy in ALS: Anna Underhill, BS - Neurology Live