# Novel Strategies for Understanding and Treating Fibrous Dysplasia

> **NIH NIH R01** · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · 2024 · $29,618

## Abstract

PROJECT SUMMARY
The overall goals of this diversity supplement are to develop the candidate’s background in biological and
translational sciences and position her to be a strong candidate for future graduate degree training. The
supplement focuses on three main approaches: Didactic research experiences, to provide exposure to a wide
breadth of ground-breaking science; hands-on laboratory experiences, to provide a realistic view of the trials and
tribulations of research as well as develop presentation skills and confidence to critically discuss scientific
questions; and direct mentoring, to provide tailored tutorials about the scientific process, molecular and cell
biology, and methods for studying genetic diseases of the skeleton. In addition, these sessions will assist with
career development and planning. The candidate will work on two aims over the course of two years. These
aims are related to the induced pluripotent stem cell (iPSC) work for compound screening in Aim 1 of the parental
R01 grant. Aim 1A: Create a series of isogenic human iPSC lines carrying the GNASR201H or GNASWT gene; Aim
1B: Use genetic modification to understand novel pathway interactions between GNAS and ACVR1 in human
iPSCs. The candidate will utilize a series of gene editing strategies we recently developed to carry out these aims.
Together, the research products of this supplement will help address one of the biggest challenges with any iPSC
model system – having robust isogenic biological replicates to demonstrate reproducibility and confirm that the
pharmacologic approaches are recapitulating the expected biological functions. The lines generated here will be
helpful for the parental Aim 1 by providing these replicates. In addition, these results will provide an opportunity
to explore a newly identified potential interaction between the GNAS and ACVR1 pathways. Elucidating these
interactions has the potential to find alternate strategic targets that could be used to manage diseases that affect
each pathway. Finally, at the completion of this experience, the candidate will be well positioned as a strong
candidate for further training in a graduate program in the sciences.

## Key facts

- **NIH application ID:** 11064244
- **Project number:** 3R01AR081336-02S1
- **Recipient organization:** UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
- **Principal Investigator:** EDWARD C HSIAO
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $29,618
- **Award type:** 3
- **Project period:** 2024-09-20 → 2027-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/11064244

## Citation

> US National Institutes of Health, RePORTER application 11064244, Novel Strategies for Understanding and Treating Fibrous Dysplasia (3R01AR081336-02S1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/11064244. Licensed CC0.

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