# Targeting the fibroblast growth factor binding protein-1 to slow degeneration of neuromuscular junctions

> **NIH NIH R01** · BROWN UNIVERSITY · 2020 · $396,250

## Abstract

Abstract: Our funded R01 is based on the discovery that FGFBP1 is associated with synapses and progressively
decreases with advancing age and in Amyotrophic Lateral Sclerosis (ALS). Specifically, we have discovered that
genetic deletion of FGFBP1 accelerates degeneration of the neuromuscular synapse during normal aging and
progression of ALS. Since FGF ligands are present and play critical functions in the brain, our findings suggest
that FGFBPs, which are master regulators of FGF ligand signaling, may play similar roles in brain synapses
harboring AD-inducing factors and with advancing age. Additionally, the increased presence of glycosylated
ECM proteins in amyloid plaques and elsewhere due to fibrosis in AD strongly indicates that changes in levels
and secretion of FGFBPs would further compromise FGF signals in AD. Thus, there is a strong rationale to posit
that FGFBPs may be necessary for FGF ligands to appropriately promote the maintenance, repair, and survival
of synapses and neurons most susceptible to AD. In this study, we propose to evaluate the role of FGFBPs in
AD in the following two aims: 1) We will determine the expression and distribution of FGFBPs in brain regions
affected with AD. 2) We will test the hypothesis that FGFBP1 plays important roles in AD pathogenesis in mouse
models of AD and in cultured neurons harboring AD-causing mutant genes.

## Key facts

- **NIH application ID:** 10121725
- **Project number:** 3R01AG055545-05S1
- **Recipient organization:** BROWN UNIVERSITY
- **Principal Investigator:** Gregorio Valdez
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $396,250
- **Award type:** 3
- **Project period:** 2017-04-15 → 2022-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10121725

## Citation

> US National Institutes of Health, RePORTER application 10121725, Targeting the fibroblast growth factor binding protein-1 to slow degeneration of neuromuscular junctions (3R01AG055545-05S1). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10121725. Licensed CC0.

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