Fabien Delerue, M.Sc., Ph.D.
Department of Genetics, Division of Discovery Science
Present Title & Affiliation
Primary Appointment
Associate Professor, Genetics, The University of Texas MD Anderson Cancer Center, Houston, TX
Director, Genetically Engineered Mouse Facility (GEMF), Genetics, The University of Texas MD Anderson Cancer Center, Houston, Texas
Dual/Joint/Adjunct Appointment
Honorary Senior Research Fellow, Biomedical Sciences, Macquarie University, Sydney, New South Wales
Research Interests
My research focuses on synthetic biology, particularly genome editing, rare genetic disorders and gene therapy. As such, I developed a genuine expertise in both genetics and neuroscience, highlighting the applicability and translational aspect of my research.
I. Disease modeling:
I worked on many projects focusing on developing and characterizing new mouse models of human conditions such as dementia, cancer or diabetes. I established a highly anticipated inducible mouse model of ALS, which recapitulates closely the hallmarks of the disease. This sophisticated mouse model has superseded previous models that lacked neuronal specific pathology. The absence of ectopic expression is based on a tightly regulated and nonleaky neuronal gene expression system that I previously established in mice. I have recently been awarded a prestigious 3Rs prize at the 18th International Society for Transgenic Technologies (ISTT) meeting. This prize is awarded for outstanding and original work that has or could have major impacts on the replacement, reduction, or refinement of the use of animals in research. Not only am I an expert in the generation of genetically engineered animal models, I am also highly experienced in a range of techniques required for the analysis of the new lines, genotypically, phenotypically, and at biochemical and physiological levels. The hundreds of mouse models that I designed, generated and characterized enabled seminal discoveries in human health and were used to develop novel therapeutic modalities.
II. Gene editing and transgenesis
In the early days of CRISPR/Cas9, I published an authoritative review of this Nobel-awarded technology. In addition, I published several reference protocols for in-vivo gene editing, and recently implemented at GEMF a highly efficient and high throughput workflow for the generation of large Knock-ins (KI) by direct electroporation of zygotes coupled to AAV infection. Using long read nanopore sequencing, I showed for the first time that on-target undesired mutations and concatemerization are substantial occurrences in CRISPR gene editing workflows, highlighting the need of a thorough quality control (QC) analysis.
III. Neuroscience:
I made seminal contributions to the understanding of pathological processes in the development of dementia and related disorders. Fundamentally changing the view of the role of the Alzheimer’s disease hallmark protein tau in normal brain physiology and disease, I was part of the Ittner team, which was the first to show that tau is present at the postsynaptic site and critically involved in glutaminergic down-stream signaling. I contributed to several significant advances in developing tau-targeted treatment of neurodegeneration. For instance, while major efforts by others focused on the development of kinase inhibitors, we took a different approach and used the small compound sodium selenate, identified in a phosphatase activation screening, to treat different tau transgenic lines, proving efficacy in vivo. Although a proof of concept at the time, sodium selenate is currently in clinical trials for different neurological conditions, including dementia. I used Artificial Intelligence-driven meta-analysis of brain gene expression to identify novel gene candidates in Alzheimer’s disease. Additional significant contributions include identifying 14-3-3θ as a potential target to treat frontotemporal dementia and amyotrophic lateral sclerosis.
IV. Rare diseases:
Generating animal models of rare genetic disorders is the first step towards any fundamental understanding of the mechanisms underlying the disease, and foundational studies characterizing these models are the condition for any pharmaceutical company to develop therapeutic programs targeting these disorders. I am heavily involved in the rare disease community, and generated several animal models of ultra-rare diseases, including a sheep model of Batten disease and mouse models of FOXG1 syndrome, macrothrombocytopenia, Canavan disease, and leukodystrophy HBSL.
Education & Training
Degree-Granting Education
| 2013 | University of Sydney, Sydney, AU, Production and characterization of new transgenic mouse models of neurodegeneration, Ph.D |
| 2002 | University of Bordeaux 1, Bordeaux 1, FR, Psychopharmacology and Pharmaceutical Research, MA |
| 2001 | University of Bordeaux 1, Bordeaux 1, FR, Cell Biology and Physiology, M.S |
Postgraduate Training
| 2013-2015 | Postdoctoral research fellow in the Dementia Research Unit, School of Medical Sciences - University of New South Wales, Sydney |
Experience & Service
Faculty Academic Appointments
Assistant Professor, Macquarie University, Sydney, 2018 - 2023
Research Associate, School of Medical Sciences, The University of New South Wales, Sydney, New South Wales, 2016 - 2018
Other Professional Positions
Manager of the Transgenic Animal Unit, School of Medical Sciences, University of New South Wales, Sydney, 2016 - 2018
Senior Technical Officer/Medical Laboratory Technician, The Brain and Mind Research Institute (University of Sydney), Sydney, 2007 - 2013
Laboratory Technician, CNS centre of F. Hoffmann-La Roche Ltd, Basel, 2002 - 2007
Extramural Institutional Committee Activities
Reviewer, GSBS student admission committee, The University of Texas MD Anderson Cancer Center, 2025 - Present
Editorial Activities
Guest editor, International Journal of Molecular Sciences (IJMS), 2024 - Present
Associate Editor, Frontiers in Genome Editing (section Genome Editing in Animals), 2022 - Present
Honors & Awards
| 2025 - Present | Multi-Year Appointment Award on the research faculty appointment track, MD Anderson Cancer Center |
| 2023 | 3Rs award: 18th International Society for Transgenic Technologies (ISTT) – Awarded for outstanding and original work that has or could have major impacts on the replacement, reduction or refinement of the use of animals in research, Houston, TX |
| 2019 | Travel grant for the fourth Oceania mouse Transgenic and Assisted Reproductive Technique symposium, Adelaide |
| 2018 | Travel grant for the first International FoxG1 symposium, San Diego, CA |
| 2015 | Travel grant for the international course on Advances in the Generation of Genetically Modified Animal Models, Montevideo, Uruguay |
| 2014 | ISTT Registration Award for the 2014 Transgenic Technology meeting, Edinburgh, Scotland |
| 2010 - 2013 | Phillip Jacoby Scholarship in Alzheimer’s Dementia Research, University of Sydney |
Professional Memberships
Grant & Contract Support
| Date: | 2025 - Present |
| Title: | MD Anderson institutional support |
| Funding Source: | MDACC |
| Role: | PI |
| Date: | 2023 - Present |
| Title: | MD Anderson Cancer Center Support Grant |
| Funding Source: | NCI |
| Role: | Key |
| ID: | P30CA016672 |
| Date: | 2023 |
| Title: | Lab and mouse handling equipment and rack washer for CAF |
| Funding Source: | Macquarie Research Infrastructure Scheme (MQRIS) |
| Role: | Collaborator |
| Date: | 2023 |
| Title: | Small-animal neurobiology platform |
| Funding Source: | Macquarie Research Infrastructure Scheme (MQRIS) |
| Role: | Collaborator |
| Date: | 2022 |
| Title: | Rapid assessment of genome editing outcomes in mice using nanopore-based adaptive sampling |
| Funding Source: | Macquarie-Mahidol Universities seed grant |
| Role: | PI |
| Date: | 2021 - 2023 |
| Title: | Novel therapeutic strategies targeting TDP-43 in Motor Neuron Disease |
| Funding Source: | Betty and John Laidlaw MND Research Prize |
| Role: | Collaborator |
| Date: | 2021 - 2023 |
| Title: | Functional analysis of long noncoding RNAs expressed in the brain |
| Funding Source: | Australian Research Council - Discovery Project |
| Role: | PI |
| Date: | 2020 - 2022 |
| Title: | Exploring technical feasibility of vertebrate pest population control with CRISPR Base-editor methods |
| Funding Source: | CSIRO-MQ collaborative research agreement |
| Role: | Collaborator |
| Date: | 2020 - 2022 |
| Title: | Develop, advance, and apply vertebrate genome editing technologies to deliver projects in biosecurity, health, and agriculture |
| Funding Source: | CSIRO-MQ collaborative research agreement |
| Role: | PI |
| Date: | 2017 - 2020 |
| Title: | Investigating the pathomechanisms of FOXG1 syndrome |
| Funding Source: | Kushagra Singha FoxG1 Research support |
| Role: | PI |
| Date: | 2017 - 2020 |
| Title: | Defining the spatial and temporal regulation of neurite branching |
| Funding Source: | ARC Discovery project |
| Role: | Collaborator |
| Date: | 2017 |
| Title: | Research Infrastructure Scheme |
| Funding Source: | Transgenic Animal Facility support staff salary |
| Role: | PI |
| Date: | 2016 |
| Title: | Research Infrastructure Scheme |
| Funding Source: | Transgenic Animal Facility support staff salary |
| Role: | PI |
| Date: | 2016 |
| Title: | Improving the frequency of targeted integration of large transgenes using CRISPR/Cas9 and long single-strand oligonucleotides (ssOligos) |
| Funding Source: | UNSW Analytical Centre Research Grant |
| Role: | PI |
| Date: | 2016 |
| Title: | Research Infrastructure Scheme |
| Funding Source: | Real-time microcirculation imaging with Laser Speckle Contrast Imaging (LSCI) |
| Role: | Collaborator |
| Date: | 2014 - 2017 |
| Title: | LRRK2 and type-1 interferon |
| Funding Source: | Michael J Fox Foundation LRRK2 challenge 2014 award |
| Role: | Collaborator |
Selected Publications
Peer-Reviewed Articles
- Liang C, Howard HR, Chen S, Choi WY, Cao S, Chien A, Zou K, Kassiou M, Delerue F, Jung HS, Park Y, Jung YM, Kwon YD, Kiang KM, Leung GK, Sugimura R, Cho AN, Lee SJ. Flash Assembloids: A Rapid Biofabrication of a Platform for Modeling Early Glioblastoma Invasion at the Glioblastoma-Brain Organoid Interfaces. Adv Healthc Mater:e04842, 2026. e-Pub 2026. PMID: 42116568.
- Chisholm CG, Bartlett R, Brown ML, Proctor EJ, Farrawell NE, Gorman J, Delerue F, Ittner LM, Vine-Perrow KL, Ecroyd H, Cashman NR, Saunders DN, McAlary L, Lum JS, Yerbury JJ. Development of a targeted BioPROTAC degrader selective for misfolded SOD1. Nat Commun 16(1):9713, 2025. e-Pub 2025. PMID: 41213972.
- Luqman, MW, Jenjaroenpun, P, Spathos, J, Shingte, N, Cummins, M, Nimsamer, P, Ittner, LM, Wongsurawat, T, Delerue, F. Long read sequencing reveals transgene concatemerization and vector sequences integration following AAV-driven electroporation of CRISPR RNP complexes in mouse zygotes. Frontiers in Genome Editing 7, 2025. e-Pub 2025. PMID: 40535113.
- Pearson, LJ, Pinyon, JL, Cederholm, J, Jonquieres, G, Bartlett, F, Vázquez-Campos, X, Delerue, F, Ittner, LM, Housley, GD. Developmental differentiation of mouse inner ear neuron subpopulations resolved with a peripherin-promoter reporter within the Grm8 locus. Scientific reports 15(1), 2025. e-Pub 2025. PMID: 40133378.
- Teboul L, Amos-Landgraf J, Benavides FJ, Birling MC, Brown SDM, Bryda E, Bunton-Stasyshyn R, Chin HJ, Crispo M, Delerue F, Dobbie M, Franklin CL, Fuchtbauer EM, Gao X, Golzio C, Haffner R, Hérault Y, Hrabe de Angelis M, Lloyd KCK, Magnuson TR, Montoliu L, Murray SA, Nam KH, Nutter LMJ, Pailhoux E, Pardo Manuel de Villena F, Peterson K, Reinholdt L, Sedlacek R, Seong JK, Shiroishi T, Smith C, Takeo T, Tinsley L, Vilotte JL, Warming S, Wells S, Whitelaw CB, Yoshiki A, Resource Association AMM, infrastructure C, consortium I, Genome Society IM, Phenotyping Consortium IM, Transgenic Technologies ISF, Resource MM, Centers R, Australia P, Resource RR, Center R, Pavlovic G. Improving laboratory animal genetic reporting: LAG-R guidelines. Nat Commun 15(1), 2024. e-Pub 2024. PMID: 38956430.
- Tan DCS, Jung S, Deng Y, Morey N, Chan G, Bongers A, Ke YD, Ittner LM, Delerue F. PLP1-Targeting Antisense Oligonucleotides Improve FOXG1 Syndrome Mice. Int J Mol Sci 25(19), 2024. e-Pub 2024. PMID: 39409184.
- Ke YD, van Hummel A, Au C, Chan G, Lee WS, van der Hoven J, Przybyla M, Deng Y, Sabale M, Morey N, Bertz J, Feiten A, Ippati S, Stevens CH, Yang S, Gladbach A, Haass NK, Kril JJ, Blair IP, Delerue F, Ittner LM. Targeting 14-3-3θ-mediated TDP-43 pathology in amyotrophic lateral sclerosis and frontotemporal dementia mice. Neuron 112(8):1249-1264.e8, 2024. e-Pub 2024. PMID: 38366598.
- Shvetcov A, Thomson S, Spathos J, Cho AN, Wilkins HM, Andrews SJ, Delerue F, Couttas TA, Issar JK, Isik F, Kaur S, Drummond E, Dobson-Stone C, Duffy SL, Rogers NM, Catchpoole D, Gold WA, Swerdlow RH, Brown DA, Finney CA. Blood-Based Transcriptomic Biomarkers Are Predictive of Neurodegeneration Rather Than Alzheimer's Disease. Int J Mol Sci 24(19), 2023. e-Pub 2023. PMID: 37834458.
- Finney CA, Delerue F, Gold WA, Brown DA, Shvetcov A. Artificial intelligence-driven meta-analysis of brain gene expression identifies novel gene candidates and a role for mitochondria in Alzheimer's disease. Comput Struct Biotechnol J 21:388-400, 2023. e-Pub 2023. PMID: 36618979.
- Morey N, Przybyla M, van der Hoven J, Ke YD, Delerue F, van Eersel J, Ittner LM. Treatment of epilepsy using a targeted p38γ kinase gene therapy. Sci Adv 8(48):eadd2577, 2022. e-Pub 2022. PMID: 36459557.
- Deng Y, Bi M, Delerue F, Forrest SL, Chan G, van der Hoven J, van Hummel A, Feiten AF, Lee S, Martinez-Valbuena I, Karl T, Kovacs GG, Morahan G, Ke YD, Ittner LM. Loss of LAMP5 interneurons drives neuronal network dysfunction in Alzheimer's disease. Acta Neuropathol 144(4):637-650, 2022. e-Pub 2022. PMID: 35780436.
- Klugmann M, Kalotay E, Delerue F, Ittner LM, Bongers A, Yu J, Morris MJ, Housley GD, Fröhlich D. Developmental delay and late onset HBSL pathology in hypomorphic Dars1M256L mice. Neurochem Res 47(7):1972-1984, 2022. e-Pub 2022. PMID: 35357600.
- Delerue F, Ittner LM. Microinjection of Zygotes for CRISPR/Cas9-Mediated Insertion of Transgenes into the Murine Rosa26 Safe Harbor. Methods Mol Biol 2495:115-128, 2022. e-Pub 2022. PMID: 35696031.
- Ittner A, Asih PR, Tan ARP, Prikas E, Bertz J, Stefanoska K, Lin Y, Volkerling AM, Ke YD, Delerue F, Ittner LM. Reduction of advanced tau-mediated memory deficits by the MAP kinase p38γ. Acta Neuropathol 140(3):279-294, 2020. e-Pub 2020. PMID: 32725265.
- Yang L, Chen Z, Stout ES, Delerue F, Ittner LM, Wilkins MR, Quinlan KGR, Crossley M. Methylation of a CGATA element inhibits binding and regulation by GATA-1. Nat Commun 11(1):2560, 2020. e-Pub 2020. PMID: 32444652.
- Brettle M, Stefen H, Djordjevic A, Fok SYY, Chan JW, van Hummel A, van der Hoven J, Przybyla M, Volkerling A, Ke YD, Delerue F, Ittner LM, Fath T. Developmental Expression of Mutant PFN1 in Motor Neurons Impacts Neuronal Growth and Motor Performance of Young and Adult Mice. Front Mol Neurosci 12:231, 2019. e-Pub 2019. PMID: 31611772.
- von Jonquieres G, Spencer ZHT, Rowlands BD, Klugmann CB, Bongers A, Harasta AE, Parley KE, Cederholm J, Teahan O, Pickford R, Delerue F, Ittner LM, Fröhlich D, McLean CA, Don AS, Schneider M, Housley GD, Rae CD, Klugmann M. Uncoupling N-acetylaspartate from brain pathology: implications for Canavan disease gene therapy. Acta Neuropathol 135(1):95-113, 2018. e-Pub 2018. PMID: 29116375.
- Tan DCS, Yao S, Ittner A, Bertz J, Ke YD, Ittner LM, Delerue F. Generation of a New Tau Knockout (tau?ex1) Line Using CRISPR/Cas9 Genome Editing in Mice. J Alzheimers Dis 62(2):571-578, 2018. e-Pub 2018. PMID: 29480201.
- Delerue F, Ittner LM. Generation of Genetically Modified Mice through the Microinjection of Oocytes. J Vis Exp(124), 2017. e-Pub 2017. PMID: 28654070.
- Bodea LG, Evans HT, Van der Jeugd A, Ittner LM, Delerue F, Kril J, Halliday G, Hodges J, Kiernan MC, Götz J. Accelerated aging exacerbates a pre-existing pathology in a tau transgenic mouse model. Aging Cell 16(2):377-386, 2017. e-Pub 2017. PMID: 28160413.
- Pleines I, Woods J, Chappaz S, Kew V, Foad N, Ballester-Beltrán J, Aurbach K, Lincetto C, Lane RM, Schevzov G, Alexander WS, Hilton DJ, Astle WJ, Downes K, Nurden P, Westbury SK, Mumford AD, Obaji SG, Collins PW, Delerue F, Ittner LM, Bryce NS, Holliday M, Lucas CA, Hardeman EC, Ouwehand WH, Gunning PW, Turro E, Tijssen MR, Kile BT. Mutations in tropomyosin 4 underlie a rare form of human macrothrombocytopenia. J Clin Invest 127(3):814-829, 2017. e-Pub 2017. PMID: 28134622.
- Frohlich D, Suchowerska AK, Spencer ZH, von Jonquieres G, Klugmann CB, Bongers A, Delerue F, Stefen H, Ittner LM, Fath T, Housley GD, Klugmann M. In vivocharacterization of the aspartyl-tRNA synthetase DARS: Homing in on the leukodystrophy HBSL. Neurobiol Dis 97(Pt A):24-35, 2017. e-Pub 2017. PMID: 27816769.
- Ittner A, Chua SW, Bertz J, Volkerling A, van der Hoven J, Gladbach A, Przybyla M, Bi M, van Hummel A, Stevens CH, Ippati S, Suh LS, Macmillan A, Sutherland G, Kril JJ, Silva AP, Mackay JP, Poljak A, Delerue F, Ke YD, Ittner LM. Site-specific phosphorylation of tau inhibits amyloid-β toxicity in Alzheimer's mice. Science 354(6314):904-908, 2016. e-Pub 2016. PMID: 27856911.
- Chandrakanthan V, Yeola A, Kwan JC, Oliver RA, Qiao Q, Kang YC, Zarzour P, Beck D, Boelen L, Unnikrishnan A, Villanueva JE, Nunez AC, Knezevic K, Palu C, Nasrallah R, Carnell M, Macmillan A, Whan R, Yu Y, Hardy P, Grey ST, Gladbach A, Delerue F, Ittner L, Mobbs R, Walkley CR, Purton LE, Ward RL, Wong JW, Hesson LB, Walsh W, Pimanda JE. PDGF-AB and 5-Azacytidine induce conversion of somatic cells into tissue-regenerative multipotent stem cells. Proc Natl Acad Sci U S A 113(16):E2306-15, 2016. e-Pub 2016. PMID: 27044077.
- Ke YD, van Hummel A, Stevens CH, Gladbach A, Ippati S, Bi M, Lee WS, Krüger S, van der Hoven J, Volkerling A, Bongers A, Halliday G, Haass NK, Kiernan M, Delerue F, Ittner LM. Short-term suppression of A315T mutant human TDP-43 expression improves functional deficits in a novel inducible transgenic mouse model of FTLD-TDP and ALS. Acta Neuropathol 130(5):661-78, 2015. e-Pub 2015. PMID: 26437864.
- Delerue F, White M, Ittner LM. Inducible, tightly regulated and non-leaky neuronal gene expression in mice. Transgenic Res 23(2):225-33, 2014. e-Pub 2014. PMID: 24214494.
- Delerue F, Sjollema G, Whittle B, Krüger S, Andrews D, Götz J. Single nucleotide variants (SNVs) define senescence-accelerated SAMP8 mice, a model of a geriatric condition. J Alzheimers Dis 36(2):349-63, 2013. e-Pub 2013. PMID: 23609764.
- Mendoza J, Lopez-Lopez C, Revel FG, Jeanneau K, Delerue F, Prinssen E, Challet E, Moreau JL, Grundschober C. Dimorphic effects of leptin on the circadian and hypocretinergic systems of mice. J Neuroendocrinol 23(1):28-38, 2011. e-Pub 2011. PMID: 20874776.
- Ittner LM, Ke YD, Delerue F, Bi M, Gladbach A, van Eersel J, Wölfing H, Chieng BC, Christie MJ, Napier IA, Eckert A, Staufenbiel M, Hardeman E, Götz J. Dendritic function of tau mediates amyloid-beta toxicity in Alzheimer's disease mouse models. Cell 142(3):387-97, 2010. e-Pub 2010. PMID: 20655099.
- van Eersel J, Ke YD, Liu X, Delerue F, Kril JJ, Götz J, Ittner LM. Sodium selenate mitigates tau pathology, neurodegeneration, and functional deficits in Alzheimer's disease models. Proc Natl Acad Sci U S A 107(31):13888-93, 2010. e-Pub 2010. PMID: 20643941.
- Ke YD, Delerue F, Gladbach A, Götz J, Ittner LM. Experimental diabetes mellitus exacerbates tau pathology in a transgenic mouse model of Alzheimer's disease. PLoS One 4(11):e7917, 2009. e-Pub 2009. PMID: 19936237.
- Leblond L, Beaufort C, Delerue F, Durkin TP. Differential roles for nicotinic and muscarinic cholinergic receptors in sustained visuo-spatial attention? A study using a 5-arm maze protocol in mice. Behav Brain Res 128(1):91-102, 2002. e-Pub 2002. PMID: 11755693.
Review Articles
- Shvetcov A, Ruitenberg MJ, Delerue F, Gold WA, Brown DA, Finney CA. The neuroprotective effects of estrogen and estrogenic compounds in spinal cord injury. Neurosci Biobehav Rev 146:105074, 2023. e-Pub 2023. PMID: 36736846.
- Delerue F, Ittner LM. Genome Editing in Mice Using CRISPR/Cas9: Achievements and Prospects. Clon Transgen(4):135, 2015. e-Pub 2015.
Professional Educational Materials
- Delerue F. Techniques d’ingénierie reproductive de la souris, manuel technique. French translation of “Reproductive engineering techniques in mice, technical manual”. Nakagata N. (3rd Edition), 2016.
Other Articles
- Klugmann M, Kalotay E, Delerue F, Ittner LM, Bongers A, Yu J, Morris MJ, Housley GD, Frohlich D Correction to: Developmental delay and late onset HBSL pathology in hypomorphic Dars1(M256L) mice. Neurochem Res 47(7):1985-1990, 2022. PMID: 35449376.
Book Chapters
- Benavides F, Delerue F, Crispo M. In: Genetically Altered Rodents. In: Rodent Quality Control: Genes and Bugs. Springer Nature (invited), 2024.
- Delerue F, Ittner LM. In: Microinjection of zygotes for CRISPR/Cas9 mediated insertion of transgenes into the murine Rosa26 safe harbour. In Applications of Genome Modulation and Editing. Springer press (invited), 2022.
- van Eersel J, Delerue F, Ittner LM, Ke YD. In: Alzheimer’s Disease and Frontotemporal Lobar Degeneration: Mouse Models. In: Neurodegenerative Diseases: Clinical Aspects, Molecular Genetics and Biomarkers for Early Diagnosis and Treatment. Springer press (invited), 2013.
Patient Reviews
CV information above last modified September 10, 2026