Researchers at Washington University School of Medicine have developed an innovative method to transform skin cells into brain neurons, enabling the study of late-onset Alzheimer's disease without the need for brain biopsies.

This groundbreaking technique utilizes cellular reprogramming to convert skin cells directly into neurons, effectively capturing age-related effects and mimicking key hallmarks of Alzheimer's, such as amyloid beta buildup and tau protein deposits.

Late-onset Alzheimer's disease, which typically manifests symptoms after the age of 65, accounts for over 95% of Alzheimer's cases, making it a critical area of research.

Senior author Andrew Yoo emphasized the complexity of sporadic, late-onset Alzheimer's due to various risk factors, highlighting the need for more effective research methods.

The study compared spheroids derived from late-onset Alzheimer's patients, inherited Alzheimer's patients, and healthy individuals, revealing significant differences in amyloid beta deposits and neuronal death that mirror clinical observations.

Findings indicated that early treatment with drugs targeting amyloid beta plaque formation significantly reduced deposits, underscoring the importance of early intervention in managing the disease.

The research also identified retrotransposable elements in the genome that change with age and contribute to late-onset Alzheimer's, suggesting new potential treatment strategies.

Inhibition of these retrotransposable elements using the anti-retroviral drug lamivudine showed promise in reducing neuronal damage and amyloid beta and tau tangles in lab-grown neuron models derived from patients.

However, lamivudine treatment was ineffective for early-onset Alzheimer's, indicating distinct molecular features between sporadic and inherited forms of the disease.

Looking ahead, the researchers plan to conduct future studies that will involve spheroids containing multiple types of brain cells, including neurons and glia, to further enhance the understanding of Alzheimer's pathology.

The findings from this research were published in the journal Science, with Andrew Yoo, PhD, serving as the senior author.