To understand disease mechanisms and ultimately develop effective therapies for AD, it is essential we apply state-of-the-art approaches to generate the next generation of more predictive animal models and make these models widely available to the AD research community.
Animal models of Alzheimer's disease (AD) have provided important insights into the pathophysiology of disease and suggested potential avenues for therapies. However, translation of these findings to the clinic has been limited. Major advances are taking place in human studies, computational approaches and animal modeling, however, that can greatly aid and accelerate this goal.
Human genetic, genomic and system biology studies pioneered by NIA-sponsored projects are beginning to reveal novel putative causative variants for late onset AD in a variety of different genes and pathways. Using this data, computational approaches are being applied to identify complex genetic networks and gene interactions relevant to AD.
Finally, recent advances in genome engineering, particularly genome editing (including CRISPR/Cas) now provide the means to efficiently and cost-effectively assess findings from human studies, validate causative variants and develop multiple animal models that will more accurately reflect the heterogeneity of human AD and also be tested for validity through biomarkers and preclinical testing.
Recent grants from the National Institute on Aging have funded several new research initiatives at JAX, including a new Alzheimer's Disease Precision Models Center (MODEL-AD) in collaboration with Indiana University. Read the full announcement.