Research
The Marro Lab investigates the molecular mechanisms underlying human brain development and disease using stem cell–based models and genetic engineering. We focus on induced pluripotent stem cells (iPSCs) and transcription factor–driven differentiation to generate neurons, glia, and brain organoids that recapitulate key aspects of neurodevelopment and neurodegeneration. By integrating genome editing technologies such as CRISPR, we model disease-relevant mutations and dissect their impact at the cellular and molecular levels. Our work spans basic mechanisms of lineage identity and maturation, as well as translational efforts to develop platforms for drug screening and regenerative approaches. We are also deeply interested in neuron–microglia interactions and the role of innate immune cells in neurological disorders.
The Team
Samuele G. Marro, PhD, is an Associate Professor of Neuroscience at the Icahn School of Medicine at Mount Sinai. He received his early training as a molecular biologist in the field of iron metabolism before shifting his focus to neuroscience. He pioneered the reprogramming of fibroblasts and other somatic cells into induced neuronal cells (iN cells), contributing to establish a foundational method for generating human neurons. His research focuses on modeling neurodevelopmental and neurodegenerative disorders using induced pluripotent stem cells (iPSCs) and gene editing technologies. He has developed widely adopted protocols for deriving human neurons and other brain cell types from iPSCs, and his lab integrates stem cell–based models and CRISPR tools to investigate disease mechanisms and explore therapeutic strategies. He is also the Director of the Stem Cell Engineering Core at the Icahn School of Medicine at Mount Sinai.
Ruiqi Hu, PhD, Assistant Scientist: Dr. Hu has co-authored multiple peer-reviewed publications employing stem cell–based approaches. She is a trained neurobiologist specializing in iPSCs, neuronal differentiation, and three-dimensional neuronal organoid cultures. Following completion of her postdoctoral training at the Icahn School of Medicine at Mount Sinai, she was promoted to Assistant Scientist. Her expertise also includes epigenetic profiling, high-throughput sequencing, neuronal imaging, and in vivo mouse work, including handling and surgical procedures.
Genevieve Smith, Research Assistant: Genevieve is a neuroscience researcher with expertise in molecular biology, gene editing, and computational analysis. She earned her BS in Neuroscience from Mercer University, where she conducted undergraduate research focused on neuroinflammation, neurodegenerative disease mechanisms, and neuronal reprogramming.
News
July 2026
New Paper Online in Stem Cell Reports
We are pleased to share that our latest study, "Induction of human pruriceptors from pluripotent stem cells via transcription factors," is now online in Stem Cell Reports.
Led by co-first authors Hisato Iriki and Ruiqi Hu, PhD, this study describes a scalable protocol to generate functional induced pruriceptors (iPruriceptors) from human iPSCs. By forcing the expression of NGN1 and ISL1 in neural crest cells, we established an in vitro platform that responds to canonical itch stimuli and exhibits in vivo-like itch pharmacology (including sensitivity to the JAK1 inhibitor ABT-317). Because this method utilizes a PiggyBac-based transfection system rather than viral vectors, it offers a simplified approach for modeling chronic pruritus and performing high-throughput drug screens.
Congratulations to Ruiqi, Hisato, and our collaborators at the Kim Lab and AbbVie on this work.
Read the open-access paper here: https://doi.org/10.1016/j.stemcr.2026.103004
April 2026
New Paper in Cell
We are excited to share that a collaborative study co-authored by the Marro Lab has been published in Cell. The work demonstrates that somatic cancer-associated variants enriched in Alzheimer's disease microglia-like cells can drive inflammatory and proliferative cellular states. Using human iPSC-derived microglia models and multi-omic approaches, the study provides new insight into how somatic mutations may contribute to neurodegeneration and Alzheimer's disease pathology.
Reference: Huang, A.Y. et al. Somatic cancer variants enriched in Alzheimer's disease microglia-like cells drive inflammatory and proliferative states. Cell (2026). doi:10.1016/j.cell.2026.03.040.
Read the paper here: https://doi.org/10.1016/j.cell.2026.03.040
January 2026
Welcome Genevieve to the Lab
We are excited to welcome Genevieve Smith to the Marro Lab as a Research Assistant. Genevieve recently earned her BS in Neuroscience from Mercer University and brings strong expertise in molecular biology, gene editing, and computational analysis. She will be working on stem cell–based models and gene-editing approaches to study neurodegenerative disease mechanisms.
July 2025
New Paper in Cell Stem Cell
We're excited to share that our latest study is now published in Cell Stem Cell! The paper reveals that TET2-mutant clonal hematopoiesis can modulate Alzheimer’s disease by promoting CNS infiltration of peripheral myeloid cells and enhancing phagocytosis. Using both UK Biobank data and iPSC-derived microglia, we demonstrate a striking TET2-specific benefit not seen with other CH drivers like DNMT3A.
Read the full paper here: https://www.sciencedirect.com/science/article/pii/S1934590925002280?via%3Dihub
Gallery
Marro Lab Publications
For a list of our publications, click the links below.
Contact
Samuele G. Marro
Institute for Regenerative Medicine
Marro Laboratory
1425 Madison Ave, 13-52
New York, NY 10029
U.S.A.