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
  • Director, Dell Pediatric Research Institute
  • Inaugural Director, Center for Rare Disease, Dell Medical School
UT Austin Dell Medical School · Department of Pediatrics · College of Natural Sciences
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Publications
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Years NIH Funded
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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), UViiVE Inc, and LEAH Labs. 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
bioRxiv Preprint 2026
“Validation and analysis of 12,000 AI-driven CAR-T designs in the Bits to Binders competitions”
PMID 41867703
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
  • Director, Dell Pediatric Research Institute 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
    UViiVE Inc
    The Agentic-first Biotech Company.
  • Active
    LEAH Labs First Gene Editing YC Company
    Fighting cancer at both ends of the leash — engineering living immune cells to fight cancer in pet dogs, with discoveries reshaping the future of human cancer therapy.

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

People — Past & Present

The Ekker Lab is built on curiosity, collaboration, and a shared commitment to turning genomic discovery into real-world impact.

Postdoctoral Fellows

Dr. Santiago Restrepo-Castillo
Dr. Santiago Restrepo-Castillo
Postdoctoral Fellow
Novel methods for mtDNA editing

Graduate Students

Adriana Morales Gomez
Adriana Morales Gomez
PhD Candidate · 2021–present
ALS and gene editing; CHCHD2/CHCHD10
Rae Mayo
PhD Student · 2023–present
Mitochondrial disease modeling in zebrafish using advanced DonorGuide gene editing
Aparna Sahu
PhD Student · 2024–present
Advances in mtDNA gene editing for zebrafish disease models
Victoria Diaz
PhD Student · 2024–present
Mitochondrial RNA transport (shared w/ Dr. Sabharwal)
MD Roushan Ali
MD Roushan Ali
PhD Student · 2025–present
Mitochondrial signaling
Smit Patel
Smit Patel
Graduate Student · 2025–present
Human mitochondrial DNA editing; hearing loss

Technicians

Sara Bohra
Sara Bohra
Research Technician
Amanda Zumbrock
Amanda Zumbrock
Research Technician

Research Support

Ron Sistrunk
Zebrafish Facility Manager
UT Austin

Undergraduates

Kyler Mitra
Kyler Mitra
Undergraduate Researcher
Zebrafish hearing loss and mtDNA variation
Rishav Sarkar
Undergraduate Researcher
Zebrafish retinal disease modeling
Ayana Bahadur
Ayana Bahadur
Undergraduate Researcher
Rare disease research
Ananya Anand
Undergraduate Researcher
Rare disease research
Anjana Reddy
Anjana Reddy
Undergraduate Researcher
Rare disease research
Danya Qadan
Danya Qadan
Undergraduate Researcher
Mitochondrial disease modeling in zebrafish
Rebecca Song
Rebecca Song
Undergraduate Researcher
Genetic compensation
Andrew Azer
Undergraduate Researcher
Jishnu Vijendran
Jishnu Vijendran
Undergraduate Researcher

Executive Assistants

Suzette Nava
Executive Assistant
Daniela Rodriguez Marty
Executive Assistant

Alumni

The Ekker Lab has trained scientists now leading labs, building companies, and advancing medicine worldwide.

Postdoctoral Alumni 21 alumni
NamePeriodPost Ekker Lab Role
Dr. Ankit SabharwalDr. Ankit Sabharwal2018–2023Faculty, UT Austin
Dr. Bibekannanda Kar2019–2023
Dr. Maarten Rotmann2019–2022Entrepreneurial Education Manager, Mayo Clinic Florida
Dr. Rena Hale2021–2022Research Associate, Mayo Clinic OoE
Dr. Annapoorna Sreedhar2021–2022Entrepreneurial Education Coordinator, Mayo Clinic Arizona
Dr. Trupti Vardam-Kaur2019–2021Assoc Tech IP Dev Mgr, Mayo Clinic Business Development
Dr. Stefan Madansingh2019–2021
Karl Clark2007–2010Associate Professor, Mayo Clinic
Christopher Pierret2008–2011Assistant Professor, Mayo Clinic
Jun Ni2007–2010
Mara Robu2003–2007Assistant Professor, St. Olaf
Darius Balciunas2001–2007Associate Professor, Temple University
Anthony Person2004–2007Scientist, R&D Systems
Michelle Knowlton2004–2007Curator, Jackson Labs
Michael Pickart2002–2005Department Head & Associate Professor, UW-Stout
Sridhar Sivasubbu2002–2005Associate Professor, IGIB New Delhi
Kevin Neff2010–2012
Alvin Ma2011–2013Assistant Professor, University of Hong Kong
Prof. Henning Schneider2009–2010 (sabbatical)Professor of Biology, DePauw University
PhD Alumni 21 alumni
NamePeriodPost Ekker Lab Role
Noriko Umemoto/Ichino2014–2020
Gabriel Martinez Galvez2016–2021Concordia University, Montreal
William Gendron2016–2021Postdoc, Dr. Barry Lab, Mayo Clinic
Hirotaka Ata2014–2019MD/PhD, Mayo Clinic; Duke University
Brandon Simone2018–2021LEAH Labs → U Penn
Jarryd Campbell2012–2016Postdoc, UMN; Scientist, Recombinetics
Stephanie Westcot2006–2016Scientific Writer, IDT; Patent Attorney, Pierce Atwood LLP
Joanna Yang Yowler2013–2017Postdoc, Mayo Clinic Florida
Margot Cousin2011–2015Research Associate, Mayo Clinic
Alaa Koleilat2016–2020Faculty, OHSU Dept. Molecular and Medical Genetics
Patrick Blackburn2012–2015Assistant Member Faculty, St. Jude's Children's Research Hospital
Greg Sindberg2014–2016Senior Medical Writer, Medtronic
Andrew Petzold2006–2010Associate Professor, Center for Learning Innovation, UMR
Brent Bill2002–2008Associate Professor, Dept. Biology, UT Tyler
Saulius Sumanas1995–2000Tenured Professor, Dept. Pathology, University of South Florida
Aidas Nasevicius1996–2001CSO, Yorktown Technologies
Ann E. Davidson1997–2004
Kamalakar Chatla2014–2015Dr. Russ Pieper Lab, UCSF
Shelly Myers2006–2007 (M.S.)
Tyler H. Koep2011–2014CSO, OneOme
MD/PhD Alumni 4 alumni
NamePeriodPost Ekker Lab Role
Victoria Bedell2008–2014Residency, University of Pennsylvania
Kirk Wangensteen2002–2007Faculty, University of Pennsylvania; GI and Hepatology, Mayo Clinic
Eleanor Chen2000–2004Assistant Professor, Dept. Pathology, University of Washington
Hyon Kim2001–2005Residency, Cincinnati Children's
Undergraduate Alumni 13 alumni
NameOutcome
Julie GuttmanMedical School (Israel)
Paul JuddGraduate School, BMBB, UMN
Jenny ShafferMedical School, Mayo Clinic
Hyon KimMD/PhD, UMN
Erin WalshMedical School, Mayo Clinic
Ken FinleyGraduate School, MCDB&G, UMN
Anna KattanGraduate School, Finland; Medical School, UMN
Cheryl SaundersGraduate School, U. Washington
Dawn DemmonGraduate School
Spencer HermansonStaff Scientist, Biotech
Paul PhelpsFulbright Student Scholar
Nicole BoczekGraduate School, Mayo Clinic
Daniel Chambers
Former Technicians 3 alumni
NamePeriodCurrent Status
Pete Strege1997–2000Scientist, Mayo Clinic
Mireya Mota–Summer 2025Graduate student, Fall 2025
Jace KleinJace Klein–Summer 2025Graduate student, Fall 2025
GREP / PREP Alumni 5 alumni
NameProgramPeriodPost Ekker Lab Role
Roberto Lopez CerveraPREP MOMSA2014–2017MD/PhD student, UMN Medical School
Jake BergrenGREP2015–2017Senior Marketing Specialist, Merck
Kyle SchaefbauerGREP2016–2017Research Technologist, Mayo Clinic
Alexandra CookGREP
Kaila SavageGREP2022
Collaborative Graduate Students & Postdocs 7 alumni
NameCollaborationPost Ekker Lab Role
Eric Mendenhallw/ Catherine VerfaillieAssistant Professor
Jeff Rossw/ Catherine VerfaillieIndustry Scientist
Melissa Ruschw/ Scott Selleck
Shannon Buckleyw/ Catherine Verfaillie
Abhishek Sohniw/ Catherine Verfaillie
Anskar LeungPostdoc w/ Catherine VerfaillieAssistant Professor, Hong Kong
Sally StringerPostdoc w/ Dr. Scott SelleckAssistant Professor, UK

The People (and Pups) Behind the Science

Ekker Lab team outing at a bowling alley

The Ekker Lab — science is a team sport

Java and Mac, Ekker Lab mascots, relaxing on the couch

Java & Mac — Chief Morale Officers 🐾

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