About

Martin Peter Carroll, MD, is an Associate Professor of Medicine (Hematology-Oncology) at the University of Pennsylvania's Perelman School of Medicine. His research focuses on the molecular biology of leukemia, with active projects investigating acute myeloid leukemia (AML) and chronic myeloid leukemia (CML). In AML, his laboratory studies oncogenic translocations and dysregulation of cellular growth mechanisms, particularly the activation of the PI3 kinase signaling pathway, which is required for the survival of primary AML cells. His work aims to understand the role of this pathway in AML and to develop targeted therapies using pathway inhibitors in preclinical models. Additionally, he explores the role of genomic instability in the progression of CML from the chronic phase to blast crisis, especially how BCR/ABL oncogene influences DNA damage response and cellular response mechanisms. Dr. Carroll's research contributes to understanding leukemia pathogenesis and developing targeted treatments for these hematologic malignancies.

Research topics

• Cancer research
• Biology
• Genetics
• Cell biology
• Immunology

Selected publications

• CRISPR-based functional genomics for dissecting therapeutic dependency in primary acute myeloid leukemia samples

  Molecular Cell · 2026-02-26

  articleOpen access

  Cancer functional genomics enables high-throughput target discovery and mechanistic investigation, yet its application has remained largely confined to mouse models and established human cancer cell lines. Direct functional interrogation of heterogeneous primary tumors offers a powerful opportunity to evaluate therapeutic targets and uncover cancer dependencies or resistance mechanisms. Here, we developed an optimized CRISPR-based platform for functional genomics in patient-derived xenograft and primary acute myeloid leukemia (AML) samples harboring diverse pathogenic mutations. Integrated in vitro and in vivo CRISPR-Cas9 knockout and CRISPR interference (CRISPRi) dropout screens validated known AML-biased targets and identified cis- regulatory elements essential for leukemic growth. Coupling pooled CRISPR perturbations with single-cell RNA sequencing (Perturb-seq) further resolved the perturbation-induced alterations in regulatory networks, cell cycle states, and cellular hierarchies in primary AML samples. Together, these studies establish a general and robust framework for leveraging CRISPR-based functional genomics to directly dissect cancer dependencies and cellular heterogeneity in primary AML patient samples. • Optimized CRISPR platform for genetic studies in primary AML samples • In vitro and in vivo CRISPR screens reveal shared and distinct AML dependencies • CRISPRi screens identify essential cis- regulatory elements in PDXs • Perturb-seq dissects regulatory networks and heterogeneity in primary AML cells Direct CRISPR functional genomics in primary patient samples holds promise for therapeutic target validation and discovery. Cao et al. develop an optimized CRISPR platform for genetic screening in primary acute myeloid leukemia samples. Integrated knockout, knockdown, and Perturb-seq screens dissect cancer vulnerabilities and cellular heterogeneity directly in patient-derived cells.

  PublisherDOI

• Supplementary Table S2 from C/EBPα Confers Dependence to Fatty Acid Anabolic Pathways and Vulnerability to Lipid Oxidative Stress–Induced Ferroptosis in <i>FLT3</i>-Mutant Leukemia

  2025-12-11

  articleOpen access

  <p>Table S2: EnrichR analysis of FLT3 gene signatures</p>

  PublisherDOI

• Supplementary Table S12 from C/EBPα Confers Dependence to Fatty Acid Anabolic Pathways and Vulnerability to Lipid Oxidative Stress–Induced Ferroptosis in <i>FLT3</i>-Mutant Leukemia

  2025-12-11

  articleOpen access

  <p>Table S12: gene signatures used to analyze RNA-seq data from patients with AML before/after GILT</p>

  PublisherDOI

• Supplementary Figures S1-S22 from C/EBPα Confers Dependence to Fatty Acid Anabolic Pathways and Vulnerability to Lipid Oxidative Stress–Induced Ferroptosis in <i>FLT3</i>-Mutant Leukemia

  2025-12-11

  articleOpen access

  <p>Supplemental figures S1-S22: Supplementary Figure S1 shows lipid metabolism dependency in FLT3-mutant AML, Supplementary Figure S2 shows anti-leukemic activity of GILT across a panel of AML PDX, Supplementary Figure S3 shows mechanisms of post-translational regulation of C/EBPα expression, Supplementary Figure S4 shows inhibition of C/EBPα phosphorylation and protein expression by FLT3i, Supplementary Figure S5 shows the implication of Ser-21 phosphorylation in post-translational regulation of C/EBPα expression, Supplementary Figure S6 shows that FLT3-ITD regulates the expression of C/EBPα and of proteins related to lipid biosynthesis in AML cell lines, Supplementary Figure S7 shows that FLT3-ITD regulates the expression of genes related to lipid biosynthesis in AML cell lines, Supplementary Figure S8 shows that C/EBPα directly regulates the transcription of lipid biosynthesis genes in AML, Supplementary Figure S9 shows the correlation between CEBPA mRNA expression and FLT3-ITD mutations, Supplementary Figure S10 shows scRNA-seq analysis from PDXTUH84, Supplementary Figure S11 shows scRNA-seq analysis from PDXTUH110, Supplementary Figure S12 shows the evolution of CEBPA_UP and FLT3-ITD_UP signatures in patients with AML treated with GILT, Supplementary Figure S13 shows that C/EBPα regulates rate-limiting lipid biosynthetic enzymes downstream of FLT3-ITD, Supplementary Figure S14 shows that C/EBPα controls lipid amount in FLT3-ITD cell lines, Supplementary Figure S15 shows that FLT3 inhibitors induce a lipid switch increasing neutral lipids dependent on C/EBPα, Supplementary Figure S16 shows that FLT3 inhibitor inhibits fatty acid synthesis fueled by glucose and glutamine, Supplementary Figure S17 shows that FLT3 inhibition decreases monounsaturated fatty acid dependent on C/EBPα, Supplementary Figure S18 shows that FLT3 inhibition increases PUFA/MUFA ratio dependent on C/EBPα, Supplementary Figure S19 shows that ferroptotic cell death induced by FLT3i is mediated by inhibition of SCD-dependent mono-unsaturated FA synthesis, Supplementary Figure S20 shows that FLT3 inhibitors unmask a vulnerability of FLT3-mutant leukemic cells to ferroptosis, Supplementary Figure S21 shows that lipid redox stress induction by GPX4 inhibition primed FLT3i activity in FLT3-ITD AML cells and Supplementary Figure S22 shows combined treatment with GILT and APR-246 in preclinical AML models in vivo.</p>

  PublisherDOI

• Supplementary Table S15 from C/EBPα Confers Dependence to Fatty Acid Anabolic Pathways and Vulnerability to Lipid Oxidative Stress–Induced Ferroptosis in <i>FLT3</i>-Mutant Leukemia

  2025-12-11

  articleOpen access

  <p>Table S15: Relative quantification by lipid class</p>

  PublisherDOI

• Supplementary Table S4 from C/EBPα Confers Dependence to Fatty Acid Anabolic Pathways and Vulnerability to Lipid Oxidative Stress–Induced Ferroptosis in <i>FLT3</i>-Mutant Leukemia

  2025-12-11

  articleOpen access

  <p>Table S4: GSEA results in FLT3-ITD PDX AML cells ex vivo-treated with QUIZ or Veh.</p>

  PublisherDOI

• Supplementary Table S1 from C/EBPα Confers Dependence to Fatty Acid Anabolic Pathways and Vulnerability to Lipid Oxidative Stress–Induced Ferroptosis in <i>FLT3</i>-Mutant Leukemia

  2025-12-11

  articleOpen access

  <p>Table S1: Transcriptome analysis of FLT3-ITD cell lines after FLT3 inhbition</p>

  PublisherDOI

• Supplementary Table S7 from C/EBPα Confers Dependence to Fatty Acid Anabolic Pathways and Vulnerability to Lipid Oxidative Stress–Induced Ferroptosis in <i>FLT3</i>-Mutant Leukemia

  2025-12-11

  articleOpen access

  <p>Table S7: Transcriptomics analysis of shCEBPA#2 versus shCTL</p>

  PublisherDOI

• Supplementary Information v.2 from C/EBPα Confers Dependence to Fatty Acid Anabolic Pathways and Vulnerability to Lipid Oxidative Stress–Induced Ferroptosis in <i>FLT3</i>-Mutant Leukemia

  2025-12-11

  articleOpen access

  <p>Supplementary materials and methods</p>

  PublisherDOI

• The LMO2-LDB1-TAL1 complex regulates transcription networks in acute myeloid leukemia

  Blood Neoplasia · 2025-11-25 · 1 citations

  articleOpen access

  Relapsed acute myeloid leukemia (relAML) remains a clinical challenge. We have shown that epigenetic heterogeneity may contribute to transcriptional dysregulation and disease progression in AML, but the specific aberrant transcriptional programs have not been identified. We analyzed molecular profiles from patient-matched diagnostic and relapse AML specimens. A subset of differentially expressed genes (DEG) that were disparate in direction of expression change identified 2 patient subtypes. We predicted that transcriptional regulators (TR) might regulate the expression patterns observed. The expression patterns of the top TR predicted for the disparate genes associated with clinical outcomes. The top TR predicted for the disparate DEG and DEG identified in a patient-derived xenograft model of relAML included members of the LIM domain only 2 - LIM domain binding 1 - TAL BHLH TF1, erythroid differentiation factor (LMO2-LDB1-TAL1) multisubunit complex (LTMC). Analysis of DepMap data identified LMO2-dependent cells with a subset highly expressing TAL1, suggesting coordinated regulation. TAL1 copurified in immunoprecipitation for LMO2 and LDB1 followed by tandem mass spectrometry analysis in HEL and K562 cells, and results from chromatin immunoprecipitation experiments suggest significant co-occupancy of TAL1 and LDB1. Loss-of-function experiments targeting LMO2, LDB1, and TAL1 in AML cell lines associated with reduced cell growth, downregulation of cell cycle genes, and a negative association with gene expression patterns observed in relapsed patients with increased TAL1 expression. Our results from primary AML specimens and functional analyses of AML cell lines supports an essential role for the LTMC in AML. Targeting the complex or downstream effectors could provide novel therapeutic considerations for a subset of patients with AML.

  PublisherDOI

Recent grants

• Understanding and Targeting Chemotherapy Resistance in Acute Myeloid Leukemia

  NIH · $3.3M · 2015–2022

• NIH Grant R01CA149566

  NIH · $2.3M · 2018

• NIH Grant R01CA100885

  NIH · $1.3M · 2010

• NIH Grant R21CA185365

  NIH · $139k · 2017

• NIH Grant I01BX000918

  NIH · 2015

Frequent coauthors

• Selina M. Luger

  161 shared

• Gerald Wertheim

  University of Pennsylvania

  113 shared

• Alexander E. Perl

  University of Pennsylvania

  101 shared

• Jennifer J.D. Morrissette

  University of Pennsylvania

  94 shared

• Adam Bagg

  University of Pennsylvania

  92 shared

• Christian Récher

  Institut universitaire du cancer de Toulouse Oncopole

  83 shared

• Robert Daber

  Invitae (United States)

  83 shared

• Pamela J. Sung

  Roswell Park Comprehensive Cancer Center

  82 shared

Labs

• Martin Peter Carroll LaboratoryPI