# Early Clonal Evolution in Acquired Aplastic Anemia

> **NIH NIH K08** · UNIVERSITY OF PENNSYLVANIA · 2021 · $88,776

## Abstract

PROJECT SUMMARY/ABSTRACT
This K08 Career Development Award proposal describes the candidate’s 5-year training program to become
an independent physician-scientist in academic hematology, with a focus on bone marrow failure 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. The strong scientific
expertise and the mentorship track record of the PI’s Mentor and Advisory Committee, together with the unique
resources and outstanding research facilities at Penn, make this an ideal environment for this K08 Award. 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:** 10393146
- **Project number:** 3K08HL132101-05S2
- **Recipient organization:** UNIVERSITY OF PENNSYLVANIA
- **Principal Investigator:** Daria Babushok
- **Activity code:** K08 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $88,776
- **Award type:** 3
- **Project period:** 2016-07-01 → 2022-03-09

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10393146

## Citation

> US National Institutes of Health, RePORTER application 10393146, Early Clonal Evolution in Acquired Aplastic Anemia (3K08HL132101-05S2). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10393146. Licensed CC0.

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