Areas of Investigation

Conklin’s team is focused on using CRISPR-related technology to treat incurable genetic diseases. By focusing on human mutations that are highly penetrant, they use patient-specific iPSCs to derive the disease tissue, and model genome surgery, prior to human clinical trials. They use whole-genome sequencing to identify common genetic polymorphisms, which can be used to selectively inactivate the disease allele with CRISPR nucleases. The diseased cell types allow them to decode the cellular signatures of disease and determine if the excision of the disease allele restores cellular functioning.

Genome surgery is a rapidly advancing field that uses state-of-the-art techniques to push the boundaries of cell and molecular biology. This laboratory uses advanced microscopy, tissue engineering, and single-cell genomics to optimize precise editing. They are also developing computational methods to select optimal CRISPR/Cas9 combinations for each disease gene. They aim to produce therapies that are safe and cost effective so they can benefit the maximal number of people. In collaboration with clinical scientists and the Innovative Genomics Institute, they are preparing large animal models and clinical-grade reagents for human clinical trials.

Lab Focus

Stem cell-derived models of human cardiovascular disease
Genome editing to study genetics, and to cure genetic diseases


Developed a novel series of assay tools (G protein chimeras) that enable high-throughput drug discovery. This method is used by 80% of major pharmaceutical companies and has contributed to the development of several approved drugs.
Engineered G protein-coupled receptors (GPCRs) called RASSLs (receptors activated by small synthetic ligands) that are unresponsive to endogenous natural hormones but can still be activated via synthetic small-molecule drugs.
Developed or contributed to various powerful, open-source bioinformatics platforms (,,, and to analyze large-scale gene-expression studies (RNA-seq).
Organized large-scale gene trapping effort with BayGenomics, inactivating over half of all mouse genes. This effort that helped inspire the international Knockout Mouse Project (KOMP).
Stem Cell Disease Modeling. In close collaboration with Dr. Yamanaka Dr. Conklin’s laboratory was able to establish new iPS cell disease models, gain new insights into iPS cell biology, and develop new tissue engineering methods.
Genome Engineering and New Experimental Technologies. Dr. Conklin’s laboratory has developed new method genome engineering in human iPS cells using the TALENs and CRISPR system. Projects also address potential approaches to therapeutic genome editing, as well as epigenetic modifications to control gene expression.


  • American Society for Clinical Investigation
  • California Academy of Science
  • Fellow
  • Assay Depot
  • Advisory Board

Professional titles

Professor, University of California, Departments of Medical Genetics and Molecular Pharmacology


  • University of California, Berkeley
  • Case Western Reserve University School of Medicine

Honors and Awards

2011 California Academy of Sciences (Fellow)
2009 Stem Cell Image Prize, California Institute for Regenerative Medicine
2008 Scientific American 50 Award
2003 American Society for Clinical Investigation (Elected)
1990 Medical Resident Research Award, NIH-NIDDKD
1988 Harry Resnick Award, Case Western Reserve School of Medicine