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The laboratory of Ngoc-Tung Tran, PhD, exploits gene editing platforms (CRISPR/Cas9) to model and develop gene therapy for monogenic disorders.

Tran Lab

The laboratory of Ngoc Tung Tran, PhD, exploits gene editing platforms (CRISPR/Cas9) to model and develop gene therapy for monogenic disorders. In collaboration with other groups at IU School of Medicine, the lab is also interested in finding novel therapeutic targets for the treatment of multiple myeloma.

The long term research goals of the Tran Lab are to understand the functional consequences of mutations that drive hematopoietic diseases/malignancies and develop novel therapeutic approaches for these diseases.

Dr. Tran conducts his research within the Hematologic Malignancies and Stem Cell Biology research program within the Department of Pediatrics Herman B Wells Center for Pediatric Research. 

Herman B Wells Center for Pediatric Research

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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.

Dr. Tran has 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 and to model blood-related disorders-driving mutations, bypassing the need for germline transmission.

Furthermore, Dr. Tran has 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 promising method may lead to a gene therapy to treat monogenic blood disorders.

Together, these key achievements create a strong foundation for 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).

Highlighted Publications

For a full list of Dr. Ngoc Tung Tran's published work, find him on PubMed.

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. View this study online.

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. View this study online.

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).

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

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

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. View this study online.

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. View this study online

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.

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. View this study online.

Faculty Research Team

59861-Tran, Ngoc Tung

Ngoc Tung Tran, PhD

Assistant Professor of Pediatrics

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