# A CENTER OF MOLECULAR HEMATOLOGY

> **NIH NIH U54** · BOSTON CHILDREN'S HOSPITAL · 2020 · $1,090,008

## Abstract

Project Summary/Abstract
In this application for an NIDDK Cooperative Hematology Specialized Core Center (CHSCC) at Boston
Children's Hospital (BCH), we seek to support the field of benign hematology research by providing crucial
resources to investigators, by bringing together interactive members who will move the field forward and recruit
new investigators to the area, by enriching the environment for trainees at all levels (undergraduate, graduate,
medical school) and by offering Pilot & Feasibility grants for highly promising innovative projects of junior
investigators. The organization and activities of the CHSCC build on an existing NIDDK-funded Center of
Excellence in Molecular Hematology that has served the Harvard Medical School community and investigators
elsewhere for 15 years. As the existing Center is the sole facility of its kind in the Harvard Medical Area, it
represents a focal point for research in benign hematology and a site for training the next generation of
investigators. The CHSCC has members at the affiliated institutions of the Harvard Medical School, and is
enriched by interactions with T32 hematology training programs at BCH and Brigham & Women's Hospital
(BWH) and a Harvard T32 training program in transfusion medicine, and with the Harvard Stem Cell Institute
(HSCI). The CHSCC is comprised of three cores, two of which center on the major animal models for
hematology research, the mouse (Core A) and zebrafish (Core B). Focus on these two systems leverages the
advantages of each, while providing the benefits of synergy from parallel developmental and genetic studies.
These cores provide state-of-the-art services for the generation of engineered mice/zebrafish, maintain critical
strains that are used in the day-to-day work of the Cores and that are distributed to investigators elsewhere,
and export methods by teaching Center members. In addition, these Cores actively develop or incorporate new
approaches, including the recently developed CRISPR/cas9 technology for genome editing. In addition, these
Cores are active in generating models of human hematologic disorders and encouraging efforts to translate
knowledge from these animal models to human. A new third core, CORE C, provides human iPS cell
reprogramming, CRISPR/Cas9 for human disease modeling, and consultation on approaches to CRISPR/Cas9
editing of mouse and zebrafish, as well as fee-for-service flow cytometry core that allows for characterization
and isolation of hematopoietic cell populations. In aggregate, the three Cores provide investigators with state-
of-the-art approaches to hematology and disease, and support innovative and disease-relevant research that
will lead to new therapeutic strategies. In addition to these cores, the CHSCC has an Administrative Core that
manages communications with the cores, advertises the capabilities of the CHSCC to investigators, and
oversees the Enrichment and Pilot & Feasibility Programs. The Enrichment Program includes many...

## Key facts

- **NIH application ID:** 9997673
- **Project number:** 5U54DK110805-05
- **Recipient organization:** BOSTON CHILDREN'S HOSPITAL
- **Principal Investigator:** STUART H ORKIN
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $1,090,008
- **Award type:** 5
- **Project period:** 2016-08-05 → 2021-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9997673

## Citation

> US National Institutes of Health, RePORTER application 9997673, A CENTER OF MOLECULAR HEMATOLOGY (5U54DK110805-05). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9997673. Licensed CC0.

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