Genome Engineering & Disease Modeling

Stephen C. Ekker Ekker Lab

From zebrafish to bedside — decoding disease one genome at a time.

We pioneer genome engineering tools, zebrafish disease models, and mitochondrial therapeutics at the intersection of discovery science and clinical translation. Based at Dell Medical School and the Department of Molecular Biosciences, The University of Texas at Austin.

Prof. Stephen C. Ekker
Principal Investigator
  • Professor of Pediatrics, Dell Medical School
  • Professor of Molecular Biosciences, College of Natural Sciences
  • Associate Dean of Innovation & Entrepreneurship, Dell Medical School
  • Associate VP of Research Translation, UT Austin
  • Inaugural Director, Center for Rare Disease, Dell Medical School
UT Austin Dell Medical School · College of Natural Sciences
0
Publications
0
Years NIH Funded
0
PubMed Indexed Papers

Advancing genomic science
from model to medicine

The Ekker Lab integrates vertebrate disease modeling, precision editing technologies, and mitochondrial biology to accelerate therapeutic development for rare and common diseases.

Zebrafish Disease Models

Over 30 years pioneering zebrafish (Danio rerio) as a vertebrate model for human disease. The lab advances tools for rigorous, reproducible zebrafish research including study design standards ("Zebrafishology"), nondestructive genotyping, and conditional genetic resources.

Precision Genome Editing

Inventors and pioneers of multiple genome editing technologies including morpholinos, TALENs, Sleeping Beauty transposon delivery, and HDR-based knock-in. Tools developed in the Ekker Lab are used by thousands of labs worldwide.

Mitochondrial DNA Disease

Developing breakthrough tools for mtDNA editing including the FusX TALE Base Editor (FusXTBE), achieving 90%+ efficiency in vivo. Modeling mtDNA disorders in zebrafish to accelerate therapeutic development for rare mitochondrial diseases.

Translational Therapeutics

Bridging discovery science to clinical application. Founded Discovery Genomics (acquired 2016), Primera Therapeutics (mitochondrial disease), and LifEngine Technologies. NIH-funded continuously for 20+ years with a track record of converting basic research into real-world impact.

Recent work from the lab

Peer-reviewed research spanning zebrafish genetics, genome editing, mitochondrial biology, and translational medicine.

medRxiv Preprint 2026
"VaaS is a Multi-Layer Hallucination Reduction Pipeline for AI-Assisted Science: Production Validation and Prospective Benchmarking"
medRxiv 2026
Nature Communications 2025
"Epigenome-wide association study of nuclear DNA methylation in relation to mitochondrial heteroplasmy"
PMID 41330919
Cells 2025
"Vascular Endothelial Growth Factor B Modulates Cardiac Functions via Ferroptosis Pathways in Post-Myocardial Infarction"
PMID 41148858
Communications Biology 2025 Review
"Zebrafishology: study design guidelines for rigorous and reproducible data using zebrafish"
PMID 40360750
Developmental Dynamics 2026
"Lineage labeling with zebrafish hand2 Cre and CreERT2 recombinase CRISPR knock-ins"
PMID 40135929
Human Gene Therapy 2024
"Unconstrained Precision Mitochondrial Genome Editing with aDdCBEs"
Zebrafish 2024
"Nondestructive Larval Genotyping of Danio rerio"

Leadership, Entrepreneurship
& Academic Service

Prof. Ekker bridges the bench and the boardroom — shaping genomics policy, training the next generation, and translating discoveries into companies and therapies.

Academic Positions

  • Professor of Pediatrics Dell Medical School, UT Austin
  • Professor of Molecular Biosciences College of Natural Sciences, UT Austin
  • Associate Dean of Innovation & Entrepreneurship Dell Medical School
  • Associate VP of Research Translation The University of Texas at Austin
  • Inaugural Director, Center for Rare Disease Dell Medical School

Entrepreneurial Ventures

  • Acquired 2016
    Discovery Genomics
    Genome editing tools and zebrafish research reagents; successfully acquired and integrated.
  • Active
    Primera Therapeutics
    Focused on mitochondrial disease therapy development using novel mtDNA editing approaches.
  • Active
    LifEngine Technologies
    Next-generation genome engineering platforms for therapeutic and agricultural applications.

Work with us

We are always looking for exceptional postdoctoral fellows, graduate students, and undergraduate researchers interested in genome engineering and disease modeling.

Postdoctoral Fellows Graduate Students Undergraduate Researchers
stephen.ekker@austin.utexas.edu

Improve this page

How can we make the Ekker Lab site better?

✓ Thanks — we'll take a look!