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ALS Mouse Models Respond Positively to Gene Therapy

Hand replacing part of DNA molecule in graphic, gene editing concept

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that currently affects about 4.42 in every 1,000,000 people worldwide. While there are a number of approved pharmaceutical treatments to slow disease progression, there is currently no cure for the disease. However, a new study published in the journal Theranostics could get researchers one step closer to finding that cure. The study was conducted by researchers at the University of California San Diego School of Medicine, who found that a gene therapy approach delayed disease onset in humanized ALS mouse models and rat models of familial ALS.

Hand replacing part of DNA molecule in graphic, gene editing concept

Precedent for Applying Gene Therapy to ALS Mouse Models

The Theranostics paper is partially based on a previous study published in 2021. In that study, the researchers used an experimental gene therapy approach known as synapsin-Caveolin-1, or SynCav1, in an attempt to prevent memory loss in a mouse model of Alzheimer’s disease. Now, the researchers have used a similar approach in ALS mouse models to determine whether the models showed signs of better motor function and longer survival after treatment.

Developing a Novel Gene Therapy

Building on the findings of the 2021 study, the research team worked with a harmless adeno-associated viral vector carrying SynCav1. The researchers injected that vector into the spinal cords of the aforementioned familial ALS mouse models. The goal was to see if the vector helped delay disease progression, preserve mobility, and maintain physical strength, including grip strength.

After careful study, the researchers found that SynCav1 protected and preserved spinal cord motor neurons. It also extended longevity in the ALS mouse models. The researchers found similar results during later experiments with a rat model of ALS, which proved extremely promising. “These data suggest that SynCav1 might serve as a novel gene therapy for neurodegenerative conditions in ALS and other forms of central nervous system disease of unknown etiology,” the authors wrote.

Treating ALS with Gene Therapy

At present, the average survival time after ALS diagnosis is between two to five years. However, with developments in gene therapy, individuals diagnosed with the disease could live longer, healthier lives with dramatically reduced symptoms. Gene therapy innovations could also prove helpful when treating other neurological conditions. “Because the neuroprotective efficacy afforded by SynCav1 occurred independent of targeting the known toxic monogenic protein (i.e., mutant hSOD1), these findings suggest that SynCav1 may serve as a novel gene therapy for other neurodegenerative conditions in addition to ALS and AD,” wrote the study’s lead author. “However, it is essential for further studies to determine the effect of SynCav1 on disease progression at later stages of the disease.”


Further research is needed to assert SynCav1 gene therapy as a valid treatment for neurodegenerative conditions like ALS. However, findings from the initial mouse model studies look promising in terms of setting up a framework for future research. Ultimately, breakthroughs in treating mouse models can be a crucial first step in developing treatments that are appropriate for humans with ALS. With further research, experts may be able to create a better quality of life for individuals in any stage of ALS progression.

Scantox is a part of Scantox, a GLP/GCP-compliant contract research organization (CRO) delivering the highest grade of Discovery, Regulatory Toxicology and CMC/Analytical services since 1977. Scantox focuses on preclinical studies related to central nervous system (CNS) diseases, rare diseases, and mental disorders. With highly predictive disease models available on site and unparalleled preclinical experience, Scantox can handle most CNS drug development needs for biopharmaceutical companies of all sizes. For more information about Scantox, visit www.scantox.com.