NIH R01 · 2025
Gene Therapy Delivery for Age-related Neurodegenerative Diseases
Project Summary/Abstract Age-related neurodegenerative disorders, such as Alzheimer’s disease and related tauopathies, are a major therapeutic challenge. There are critical brain regions implicated in disease pathogenesis and overtime the whole or a large portion of the brain is affected. Therefore, both a focal and broad brain treatment approach is needed to achieve long-term therapeutic benefit. Gene therapy is the use of genetic material to target disease etiology. Viral vectors are used to deliver therapeutic genes to cells to provide long-lasting intervention from a single treatment. Recombinant adeno-associated virus (rAAV) vectors are highly used due to their efficient gene transfer,…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.