The median survival time after diagnosis of the disease is two to five years.
A study of University of California San Diego School of Medicine (United States) concludes that a gene therapydeveloped in said center, delayed “appreciably” the appearance of the Amyotrophic Lateral Sclerosis (ELA) familial in humanized mouse and rat models, also increasing their life expectancy.
Familial ALS is an inherited form of the disease that runs in families. The researchers of the study, published in the journal ‘Theranostics‘, remember that ALS is a neurodegenerative disease characterized by the progressive loss of motor neurons in the brain and spinal cord responsible for voluntary movements and muscle control. Most cases of ALS are sporadic, of unknown cause, although environmental and genetic factors may play a role.
In earlier research, lead author Brian P. Head, associate professor in the Department of Anesthesiology at UC San Diego School of Medicine, and colleagues had bred a mouse model genetically engineered to express a neuroprotective protein called caveolin- 1 with a transgenic mouse model of ALS. The double transgenic model had better motor function and longer survival.
The latest work involved injecting a harmless adeno-associated viral vector carrying synapsin-caveolin-1 cDNA (AAV9-SynCav1) into the spinal cords of mice with familial ALS to see if it delayed the disease progression and preserved physical strength and mobility.
The researchers found that SynCav1 protected and preserved motor neurons in the spinal cord and prolonged the longevity of mice. Subsequent experiments with a rat model of ALS produced similar results.
Novel gene therapy for neurodegenerative conditions
“These data suggest that SynCav1 could serve as a novel gene therapy for ALS neurodegenerative conditions and other forms of central nervous system disease of unknown etiology,” wrote the authors, who have advocated further study.
The Theranostics paper follows up on a study published in 2021 in which Head and colleagues used SynCav1 gene therapy to prevent cancer. learning and memory loss in a mouse model of Alzheimer’s disease, a key step in testing the approach in humans with the neurodegenerative disease.
“Since the neuroprotective efficacy of SynCav1 is independent of the known toxic monogenic protein (ie, mutant hSOD1), these results suggest that SynCav1 may serve as a new gene therapy for other neurodegenerative diseases, in addition to ALS and Alzheimer’s.” , according to Head. “However, it is essential that further studies be carried out to determine the effect of SynCav1 on disease progression in later stages of it,” he adds.
The incidence of ALS is approximately 3 to 5 per 100,000 people worldwide. The disease affects about 18,000 people in the United States. Current approved pharmaceutical treatments, such as Rilutek and Radicava, can slow disease progression and improve quality of life, but there is no cure. Median survival time after diagnosis is two to five years.
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