The laboratory of Stephen C. Ekker, Ph.D., develops and deploys new precision molecular genetic tools using the zebrafish as the pioneering animal model to enable a better understanding of the human genome. From gene editing to genome engineering, these genome writing systems empower researchers to better understand the underlying mechanisms of disease while also serving as the basis for new therapies.
The Ekker lab established morpholinos as the pioneering sequence-specific knockdown technology for vertebrate functional genomics applications using the zebrafish two decades ago. In parallel, they developed vertebrate transposon tools including protein trap gene-breaking vectors to generate a collection of more than 1,000 molecularly characterized and revertible mutant zebrafish lines. This was the first engineered revertible alleles in any organism outside the mouse.
Dr. Ekker and colleagues deployed transposons in diverse application areas including human T cells, zebrafish and mice. Custom restriction endonucleases offer an additional approach to targeted modification using genome editing tools. Recent gene editing advances have enabled new F0 screening work (PreMA alleles), targeted knockins for the extremely effective gene break insertional mutagen using the GeneWeld method, and new programmable deletions in the mitochondrial genome.
Work in Dr. Ekker's lab continues to develop the science behind these new engineering toolkits, working with laboratories that study rat, pig, mouse, nematode and fly, in addition to colleagues who work in human cells and zebrafish.
- Gene editing and genome engineering. Dr. Ekker's research team continues to develop innovative approaches for precise changes of the genomes with an emphasis on functional genomics of understudied loci.
- Novel therapies. Research in Dr. Ekker's lab harnesses new gene editing methods for cutting-edge therapeutic approaches, including both nuclear- and mitochondria-encoded genetic diseases.
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Significance to patient care
Dr. Ekker's research explores novel options for using pioneering gene editing methods to address unmet patient needs, with an emphasis on those facing more than 4,000 examples of rare diseases with a major genetic component.