# Early Clonal Evolution in Acquired Aplastic Anemia

> **NIH NIH K08** · UNIVERSITY OF PENNSYLVANIA · 2020 · $54,000

## Abstract

PROJECT SUMMARY/ABSTRACT
This is a Supplement for the Mentored Clinical Scientist Development Award (K08- HL132101). K08-
HL132101 was funded on 07/01/2016 and describes the candidate’s 5-year training program to become an
independent physician-scientist in academic hematology, with a focus on bone marrow failure (BMF)
syndromes. The Principal Investigator (PI) has completed M.D. and Ph.D. Degrees at the University of
Pennsylvania (Penn), followed by residency training in Internal Medicine at the Massachusetts General
Hospital, fellowship training in Hematology-Oncology at Penn, and is currently an Assistant Professor in
Medicine at the University of Pennsylvania. Through the 5-year career development plan of the parent K08
award, the PI will expand her knowledge and research skills in hematopoiesis, stem cell biology, and
immunology in order to develop an independent research program studying clonal hematopoiesis in acquired
aplastic anemia (aAA). The PI will be mentored by Dr. Peter Klein, an expert on hematopoiesis, as well as
the Advisory Committee who will foster PI’s scientific and career development.
The proposed research focuses on aAA, a life-threatening blood disease, affecting children and adults,
caused by immune destruction of early hematopoietic cells. Clonal evolution to leukemia is a common
complication, with no effective prevention strategy available. Emerging data indicate that up to a quarter of
aAA patients acquire somatic mutations, and may be at a greater risk of malignant transformation.
Therefore, understanding clonal hematopoiesis in aAA is important to allow for early detection and improved
therapies. The objective of the K08 award is to characterize the full spectrum of clonal hematopoiesis in aAA
and to dissect the mechanisms driving emergence of clones. This will be accomplished in three specific
aims: 1) Characterize the landscape and prevalence of somatic mutations in the bone marrow of aAA
patients using an unbiased genetic analysis, 2) Identify genomic biomarkers of response to therapy and
disease outcomes, and 3) Characterize the function of candidate driver mutations in hematopoiesis. The
successful completion of this project is expected to have a sustained and lasting impact in the field by
providing an understanding of clinically-relevant somatic alterations in aAA, which could serve as genetic
biomarkers and targets for new personalized therapies. Unfortunately, the PI’s scientific progress was
impacted by a critical life event during the award period. This administrative supplement will provide critical
funding to offset the costs of technician and supplies to allow the PI to accelerate the pace of experiments to
continue to move forward in her career and establish research independence. This research program,
performed in the context of a comprehensive career development plan, will support the PI’s career transition
to become an independent physician-scientist in Hematology.

## Key facts

- **NIH application ID:** 10142254
- **Project number:** 3K08HL132101-05S1
- **Recipient organization:** UNIVERSITY OF PENNSYLVANIA
- **Principal Investigator:** Daria Babushok
- **Activity code:** K08 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $54,000
- **Award type:** 3
- **Project period:** 2016-07-01 → 2022-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10142254

## Citation

> US National Institutes of Health, RePORTER application 10142254, Early Clonal Evolution in Acquired Aplastic Anemia (3K08HL132101-05S1). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10142254. Licensed CC0.

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