# Novel Gene Editing Approaches for Hemoglobinopathies

> **NIH NIH R01** · FRED HUTCHINSON CANCER RESEARCH CENTER · 2020 · $921,786

## Abstract

Disorders of hemoglobin production are the most common inherited disorders of hematopoiesis, and are a
cause of immense human morbidity and economic loss across the globe. Recent advances in gene-editing
technology have demonstrated the potential to directly manipulate the endogenous globin locus in human
hematopoietic stem and progenitor cells. However, significant barriers remain for translation of gene editing as
an effective therapeutic approach to disorders of globin production, including 1) definition of gene-edited globin
locus architectures that support high levels of non-sickling globin production, 2) maintenance of multi-lineage
reconstitution capability of gene-edited HSPC, and 3) development of methods for efficient engraftment and
selection of gene-edited HPSC. In this application, we propose two coordinated specific aims that collectively
address these barriers. Specific Aim 1 will utilize recently published methods for efficient HSPC gene editing
to define globin locus architecture(s) that support high levels of erythroid specific globin production and that
enable post-engraftment selection of gene-edited cells. Specific Aim 2 will evaluate engraftment and selection
protocols for gene-edited HSPC in a clinically relevant macaque autologous transplant model. Collectively, the
proposed studies will define an optimized globin gene editing procedure along with a IND-enabling pre-clinical
data set that will serve as a foundation for translation of therapeutic gene editing for disorders of β-globin
synthesis to phase I clinical testing in humans.

## Key facts

- **NIH application ID:** 9850621
- **Project number:** 5R01HL136135-04
- **Recipient organization:** FRED HUTCHINSON CANCER RESEARCH CENTER
- **Principal Investigator:** HANS-PETER KIEM
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $921,786
- **Award type:** 5
- **Project period:** 2017-01-17 → 2020-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9850621

## Citation

> US National Institutes of Health, RePORTER application 9850621, Novel Gene Editing Approaches for Hemoglobinopathies (5R01HL136135-04). Retrieved via AI Analytics 2026-05-28 from https://api.ai-analytics.org/grant/nih/9850621. Licensed CC0.

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