# Improving transfusion therapy for patients with sickle cell disease with pluripotent stem cell-derived red cells

> **NIH NIH U01** · CHILDREN'S HOSP OF PHILADELPHIA · 2021 · $1,264,180

## Abstract

ABSTRACT: OVERALL
Red blood cell transfusion remains a life-saving therapy for patients with sickle cell disease (SCD). A major
problem is the high rate of alloimmunization (antibody formation against transfused red cells) that occurs in
transfused patients with SCD. Genetic diversity in blood group antigens in patients of African descent
compared to the primarily European-based donor pool contributes to this high incidence and complexity of
antibodies found in patients with SCD. Finding compatible red blood cell (RBC) units is often complicated by a
lack of rare reagent RBCs to properly identify these complex antibody specificities. This complication delays
care, increases costs, and makes transfusion therapy impossible for some patients. The ultimate goal of this
proposal is to use human induced pluripotent stem cells (iPSCs) to produce standard and reliable red blood
cell (RBC) reagents to resolve this major problem. We have designed a panel of iPSCs genetically engineered
to express unique combinations of blood group antigens (customized iPSCs) that are difficult or virtually
impossible to find in donor RBCs. RBC reagents produced from these customized iPSCs will provide the
means to streamline and standardize antibody identification in alloimmunized patients, and ultimately can be
used as “universal” donor cells for future therapeutic applications. Our efforts will address several existing
challenges that include: i. insufficient or no living blood donors expressing the combinations of blood group
antigens needed to resolve the complex antibody specificities in patients with SCD, ii. lack of iPSC
differentiation protocols to produce definitive, adult-type RBCs without the use of serum or stromal cells, and iii.
the prohibitively expensive manufacturing costs of iPSC-derived RBCs. In this U01 application, we propose
three integrated Projects from a group of highly collaborative investigators with expertise in areas that can
address all three challenges and will drive the field forward by providing innovative solutions to these current
obstacles.

## Key facts

- **NIH application ID:** 10181018
- **Project number:** 5U01HL134696-06
- **Recipient organization:** CHILDREN'S HOSP OF PHILADELPHIA
- **Principal Investigator:** STELLA T CHOU
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $1,264,180
- **Award type:** 5
- **Project period:** 2016-09-15 → 2023-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10181018

## Citation

> US National Institutes of Health, RePORTER application 10181018, Improving transfusion therapy for patients with sickle cell disease with pluripotent stem cell-derived red cells (5U01HL134696-06). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10181018. Licensed CC0.

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