# Closing the gap between structural biology and translational science for amyloid diseases

> **NIH NIH DP2** · UT SOUTHWESTERN MEDICAL CENTER · 2021 · $1,437,088

## Abstract

PROJECT SUMMARY/ABSTRACT
The recent wave of cryo-electron microscopy (cryo-EM) studies on amyloid fibrils has highlighted the complexity
of protein deposition amongst patients of amyloid diseases. A convincing example is found in tau, a microtubule
binding protein whose pathological aggregation causes tauopathies. Each of these tauopathies seems to be
associated with a particular structural assembly, suggesting causality. Similar to tauopathies, other amyloid
diseases manifest differential prognosis, onset, and symptomatology. This is the case of TDP-43, α-synuclein,
β-amyloid, or transthyretin (TTR), to name a few. We hypothesize that these phenotypical differences are driven
by the formation of various structural assemblies, similar to what is found in tau. As a consequence, the detection
of these disease-specific assemblies in a timely manner could ensure proper diagnosis and treatment.
We aim to map the structural spectrum of amyloid fibrils in an amyloid disease model using cryo-EM and a co-
culture system to be developed in our laboratory. Using the obtained structural information, our laboratory will
design structure-specific peptides for the detection and inhibition of amyloid fibrils in cells, mice, and patient-
derived samples. If successful, our study will serve as a launching platform for the development of personalized
structure-based diagnostics and therapeutics for amyloid diseases.

## Key facts

- **NIH application ID:** 10242433
- **Project number:** 1DP2HL163810-01
- **Recipient organization:** UT SOUTHWESTERN MEDICAL CENTER
- **Principal Investigator:** Lorena Saelices Gomez
- **Activity code:** DP2 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $1,437,088
- **Award type:** 1
- **Project period:** 2021-09-01 → 2024-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10242433

## Citation

> US National Institutes of Health, RePORTER application 10242433, Closing the gap between structural biology and translational science for amyloid diseases (1DP2HL163810-01). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10242433. Licensed CC0.

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