# Stem cells for therapeutics discovery in genetic blood disorders

> **NIH NIH U01** · BOSTON CHILDREN'S HOSPITAL · 2021 · $1,223,081

## Abstract

Abstract
Under the auspices of the Progenitor Cell Biology Consortium (PCBC), our hub at
Boston Children's Hospital used unbiased chemical screens in zebrafish and human
induced Pluripotent Stem (iPS) Cells to identify two drugs that rescue the hematopoietic
defects in Diamond Blackfan Anemia, a genetic bone marrow failure syndrome that lacks
adequate pharmacologic or cell-based treatments. In the initial years of the next phase—
the Progenitor Cell Translation Consortium—we propose to initiate clinical trials with
these agents, and to establish a comprehensive center that during the subsequent years
will develop new drugs, progenitor-based cell therapies, and gene therapies for
additional genetic blood diseases, including Shwachman Diamond Syndrome, Fanconi
Anemia, Dyskeratosis Congenita, primary immune deficiency, and hemoglobinopathy.
We will build competency for both internal discovery and clinical translation, as well as
Core facilities and generic platforms that will invite collaborations from external
investigators, thereby serving the broader mission of the PCTC. Success in our aims will
address considerable unmet needs for both the investigation of fundamental
mechanisms of genetic blood disease and the development of novel therapeutics for
conditions that while individually rare, are collectively a major challenge for hematology.
The blood affords considerable advantages as a discovery system, given that analytic
reagents and molecular catalogues of blood lineages abound; the high probability of
success for blood diseases will establish a powerful proof-of-principle for leveraging
human iPS cell models in clinical translation for other diseases.

## Key facts

- **NIH application ID:** 10188598
- **Project number:** 5U01HL134812-06
- **Recipient organization:** BOSTON CHILDREN'S HOSPITAL
- **Principal Investigator:** George Q Daley
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $1,223,081
- **Award type:** 5
- **Project period:** 2016-09-23 → 2023-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10188598

## Citation

> US National Institutes of Health, RePORTER application 10188598, Stem cells for therapeutics discovery in genetic blood disorders (5U01HL134812-06). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10188598. Licensed CC0.

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