Skip to main content
U.S. flag

An official website of the United States government

FTD Basic Research: Establish new animal and cellular models (Milestone 4.Q)

In Progress

Timeline Start - End

2016 - 2026

Research Implementation Area

Translational Tools, Infrastructure, and Capabilities

AD-Related Dementias Focus

Advance understanding of FTD and identify therapeutic targets through the creation, validation, and use of pre-clinical and translational tools and resources.

Success Criteria

  • At least three new animal and/or cell-based models that replicate key aspects human FTD.
  • Develop and/or validate at least two in vivo functional assays for FTD translational research, including with endpoints in animal models that are relevant for FTD biology and/or clinical outcomes.
  • One new resource that harmonizes critical FTD datasets across disease stages for data sharing to enable translational research.

Summary of Key Accomplishments

In addition to supporting the development of better FTD disease models through our standard grant mechanisms such as NS097273 which uncovered a potential mechanism contributing to neurodegeneration by the FTD-linked mutation in the C9ORF72 gene, the NIH also released a targeted call for applications in this area in 2019 (PAR-19-167).

Examples of funded awards from this program include the characterization of a non-human primate model found to have a spontaneous mutation identical to that found in people with FTD , the development of mouse models that more completely mimic human disease at the genetic level, and the development of "organs-on-a-chip" that put cells grown in the lab in more biologically-relevant conditions with the potential for informing future clinical trials. Future work is progressing to develop models that better inform on specific disease mechanisms, biomarker discovery, and therapeutic target identification in FTD, while model generation is also being expanded to include computational (i.e., big data) models.

The key accomplishments summary is current as of November 2022.

Accomplishments/Implementation Activities

Funding Initiatives

Research Programs and Resources

Research Highlights

Relevant Recommendations

Related Milestones

Search all Research Categories

An official website of the National Institutes of Health