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Scientists in the United Kingdom have achieved a breakthrough using lab-made “mini-brains” to study a gene associated with neurodegenerative diseases like amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and Parkinson’s disease. This research may lead to the development of innovative detection methods and treatments for these diseases, potentially addressing them before symptoms manifest.

Angiogenin crucial in stem cells differentiation

Researchers from the University of Bath highlight the crucial role of the Angiogenin (ANG) gene in the normal development of undifferentiated stem cells into specialized nerve cells. However, complications arise with a mutated version of the gene, leading to disruptions in this developmental process.

Mutated ANG induces prolonged maintenance of stem cells in their initial state, delaying the differentiation process. Lab tests reveal significant neurodevelopmental abnormalities in adult nerve cells due to this slowed differentiation.

Dr. Vasanta Subramanian, research leader from the Department of Life Sciences, indicates that nerve-cell degeneration could be triggered by early developmental defects.

In a prior study, researchers found that the healthy form of the ANG gene safeguards nerve cells from damage and degeneration. Conversely, the mutated gene increases susceptibility to stress, accelerating cell death. According to Dr. Subramanian the discovery enhances understanding of Angiogenin’s role in protecting against age-related diseases.

Brains with ANG mutations had neurodevelopmental defects

The research team conducted a study on a family with frontotemporal dementia and ALS. Genetic testing revealed Angiogenin mutations in some family members. During the study, mini-brains were grown for each member in a lab and those with ANG mutations showed significant neurodevelopmental defects, serving as valuable disease models for drug screening.

Dr. Subramanian suggests that subtle developmental defects may contribute to disease susceptibility. He envisions a future where individuals at risk are identified, screened for genetic mutations, and provided with early-intervention gene therapy to address these defects, indicating a proactive approach to disease prevention and treatment.

According to Dr. Subramanian more research is necessary to elucidate ANG’s cell protection mechanisms and its role in stem cells. The National Centre for the Replacement, Refinement and Reduction of Animals in Research (NC3Rs), BRACE, and the Wellcome Trust VIP award provided funding for the study.