59861-Tran, Ngoc Tung

Ngoc Tung Tran, PhD

Assistant Professor of Pediatrics

R4 166

Indianapolis, IN


After finishing the B.S from Hanoi University of Sciences, Hanoi, Vietnam, Dr. Tran went to KAIST, South of Korea, to pursuit his Ph.D. in stem cell biology. Then, as a postdoc, he joined Dr. Xinyang Zhao’s group, the University of Alabama at Birmingham, Alabama, U.S, to work intensively on cancer biology, biochemistry, and hematopoiesis. Inspired by the thriving of the CRISPR/Cas9 system, he moved to Klaus Rajewsky’s laboratory, Max Delbrück Center for Molecular Medicine, Berlin, Germany, to study hematopoiesis, lymphomagenesis, and immunity. Here, he has developed an efficient system to knock in and knock out genes in hematopoietic stem/progenitor cells (HSPCs) and induced pluripotent stem cells (iPSCs). This system can be broadly exploited to both modeling and developing gene therapy for blood disorders.
Dr. Tran joined the Department of Pediatrics at Indiana University in May 2021 to develop his independent research program aiming to study hematopoietic malignancies. His ultimate goal is to develop gene therapy and therapeutic approaches for blood disorders.

Key Publications

1.     Hairui Su, Ming Jiang, Chamara Senevirathne, Srinivas Aluri, Tuo Zhang, Han Guo, Juliana Xavier-Ferrucio, Shuiling Jin, Ngoc-Tung Tran, Szu-Mam Liu, Chiao-Wang Sun, Yongxia Zhu, Qing Zhao, Yuling Chen, LouAnn Cable, Yudao Shen, Jing Liu, Cheng-Kui Qu, Xiaosi Han, Christopher A. Klug, Ravi Bhatia, Yabing Chen, Stephen D. Nimer, Y. George Zheng, Camelia Iancu-Rubin, Jian Jin, Haiteng Deng, Diane S. Krause, Jenny Xiang, Amit Verma*, Minkui Luo*, and Xinyang Zhao*. Methylation of Dual Specificity Phosphatase 4 Controls Cell Differentiation. Cell Reports 2021 (in press)

2.     Ngoc-Tung Tran, Robin Graf, Annika Wulf-Goldenberg, Maria Stecklum, Gabriele Strauß, Ralf Kühn, Klaus Rajewsky, and Van Trung Chu. CRISPR/Cas9-mediated ELANE mutation correction in hematopoietic stem/progenitor cells to treat Severe Congenital Neutropenia. (Molecular Therapy Vol. 28 No 12 December 2020. https://doi.org/10.1016/j.ymthe.2020.08.004).

3.     Ngoc Tung Tran, Janine Trombke, Klaus Rajewsky, and Van Trung Chu. Protocol for Efficient CRISPR/Cas9/AAV-Mediated Homologous Recombination in Mouse Hematopoietic Stem and Progenitor Cells. STAR Protocol April 2020, DOI: 10.1016/j.xpro.2020.100028.

4.     Ngoc-Tung Tran, Thomas Sommermann, Janine Trombke, Robin Graf, Jenniffer Pempe, Kerstin Petsch, Ralf Kuehn, Klaus Rajewsky, and Van Trung Chu. Efficient homologous recombination in mouse hematopoietic stem/progenitor cells. (Cell Reports 28, 3510–3522. https://doi.org/10.1016/j.celrep.2019.08.065)

5.     Thi Thanh Huong Le*, Ngoc-Tung Tran*, Thi Mai Lan Dao, Dinh Dung Nguyen, Huy Duong Do, Thi Lien Ha, Ralf Kuehn, Thanh Liem Nguyen, Klaus Rajewsky, Van Trung Chu. Efficient and precise CRISPR/Cas9-mediated MECP2 modifications: a potential therapeutic approach for Vietnamese RTT patients. (Front. Genet. DOI: 10.3389/fgene.2019.00625). * Co-first author

6.     Ngoc-Tung Tran, Sanum Bashir, Xun Li, Jana Rossius, Van T. Chu, Klaus Rajewsky, Ralf Ku¨hn. Enhancement of precise gene editing by the association of Cas9 with homologous recombination factors. (Front. Genet. (2019). doi: 10.3389/fgene.2019.00365.)

7.     Jin S, Su H, Tran NT, Song J, Lu SS, Li Y, Huang S, Abdel-Wahab O, Liu Y, Zhao X. Splicing factor SF3B1K700E mutant dysregulates erythroid differentiation via aberrant alternative splicing of transcription factor TAL1. PLoS One. 2017 May 18;12(5): e0175523. doi: 10.1371/journal.pone.0175523. eCollection 2017.

8.     Chu VT, Graf R, Wirtz T, Weber T, Favret J, Li X, Petsch K, Tran NT, Sieweke MH, Berek C, Kühn R, Rajewsky K. Efficient CRISPR-mediated mutagenesis in primary immune cells using CrispRGold and a C57BL/6 Cas9 transgenic mouse line. Proc Natl Acad Sci U S A. 2016 Nov 1;113(44):12514-12519. Epub 2016 Oct 11.

9.     Ngoc-Tung Tran, Hairui Su, Li Zhang, Alireza Khodadadi-Jamayran, Shan Lin, Dewang Zhou, Kevin Pawlik, Tim Townes, James C. Mulloy, and Xinyang Zhao. Long noncoding RNA (AS-RBM15) controls RBM15 translation to promote megakaryocyte differentiation. EMBO Rep. 2016 Jun;17(6):887-900. doi: 10.15252/embr.201541970. Epub 2016 Apr 26.

10.  Li Zhang*, Ngoc-Tung Tran*, Hairui Su, Rui Wang, Yuheng Lu, Haiping Tang, Ailan Guo, Alireza Khodadadi-Jamayran, Dewang Zhou, Kun Qian, Wenping Zhou, Todd Hricik, Jocelyn Côté, Xiaosi Han, Suparna Laha, Omar Abdel-Wahab, Ross Levine, Glen Raffel, Yanyan Liu, Dongquan Chen, Haitao Li, Tim Townes, Hengbin Wang, Haiteng Deng, Y. George Zheng, Christina Leslie, Minkui Luo, and Xinyang Zhao. Crosstalk between PRMT1-mediated methylation and ubiquitylation on RBM15 controls RNA splicing. Elife. 2015 Nov 17;4. pii: e07938. doi: 10.7554/eLife.07938. * Co-first author.

11.  Sang-Wook Park; Jihoon Kim; Jong-Lyul Park; Ji-Yun Ko; Ilkyun  Im; Hyo-Sang Do; Ngoc-Tung Tran; Hyemin Kim; Sang-Hun  Lee; Yong Sung  Kim; Yee Sook Cho; Dong Ryul Lee; Yong-Mahn Han. Variable allelic expression of imprinted genes in human pluripotent stem cells during differentiation into specialized cell types in vitro. Biochem Biophys Res Commun. 2014 Apr 4;446(2):493-8.

12.  Tran NT, Trinh QM, Lee GM, and Han YM. Efficient Differentiation of Human Pluripotent Stem Cells into Mesenchymal Stem Cells by Modulating Intracellular Signaling Pathways in a Feeder/Serum-Free System. Stem Cells Dev. 2012 May 1; 21(7):1165-75.




Titles & Appointments

  • Assistant Professor of Pediatrics
  • Assistant Professor of Microbiology & Immunology
  • Assistant Professor of Microbiology and Immunology
  • Education
    2012 PhD Korea Advanced Institute of Science and Technology
    2006 BS Vietnam National University
  • Research

    My long-term research goals are to understand the functional consequences of mutations driving hematopoietic diseases/malignancies and develop novel therapeutic approaches for these diseases.

    Hematopoietic stem cells are self-renewing and multipotent cells that give rise to all types of blood cells in the body. Mutations in single genes might cause severe blood-related diseases. Understanding the functional consequences of these mutations is crucial for therapy development. I have developed an efficient system to knock in and knock out genes in hematopoietic stem/progenitor cells (HSPCs). This is an ideal system to study in vivo functions of novel genes in hematopoiesis as well as to model blood-related disorders-driving mutations, bypassing the need for germline transmission. Furthermore, I have established a CRISPR/Cas9-based system to efficiently repair mutations in human HSPCs at multiple disease-relevant loci (HBB, FANCG, and ELANE) with minimal off-target activities and unwanted on-target mutations. This will hopefully lead to a promising gene therapy to treat monogenic blood disorders.

    These key achievements create a strong foundation for my future research programs that include both clinically oriented (Modeling and developing gene therapy for hematopoietic disorders) and basic research (Exploring novel in vivo functions of genes in hematopoiesis and lymphomagenesis).

  • Professional Organizations
    American Society of Gene and Cell Therapy
    American Society of Hematology

Research Labs

Faculty research at IU School of Medicine is transforming health. Details about the medical research being conducted in faculty labs throughout IU School of Medicine are available in the Research section of this site.

Faculty Labs