Bruce Conklin’s team aims to cure genetic diseases using state-of-the-art genome-engineering technology. This involves using patient-specific induced pluripotent stem (iPS) cells to derive tissue from patients who carry disease mutations that could benefit from therapeutic genome editing with CRISPR. Conklin’s team focuses on modeling diseases in iPS cells, then testing the effect of genome editing. Their proof-of-concept studies are aimed at rare mutations with the highest chance of a measurable clinical impact, with the goal to expand to more common diseases. Conklin’s group hopes to produce therapies that are safe and cost-effective so they can benefit the maximum number of people.

Disease Areas

Alpha Thalassemia
Best Disease
Heart Failure
Genetic Forms of Cardiomyopathy
Motor Neuron Diseases (Charcot-Marie-Tooth and ALS)

Areas of Expertise

Cardiomyocyte Biology
CRISPR and Genome Editing
Human Genetics
Molecular Pharmacology
Motor Neuron Biology
Stem Cells and iPS Cells
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Lab Focus

Using human genetics to understand disease mechanisms.
Creating stem cell-derived models of human genetic disease.
Leveraging genome-editing technology to study and cure genetic diseases.

Research Impact

Stem cell biology and therapeutic genome editing with CRISPR are both rapidly developing fields that will drive a new level of investigation into diverse cell types associated with genetic diseases, including cardiomyocytes, motor neurons, and retinal pigment epithelial cells. Only by understanding the basic cellular and molecular physiology of these cells can scientists meet the challenges that lie ahead.

Conklin’s lab investigates how genetic mutations affect the formation of different cell types to probe the 3D architecture and epigenetic state of the genes of interest. In addition, as the team learns to orchestrate precise genomic repair, they are gaining a better understanding of how DNA repair machinery differs in each cell type. Furthermore, they perform genome engineering in authentic cellular models to provide mechanistic insights into disease processes and the potential for functional recovery.

 

Professional Titles

Senior Investigator, Gladstone Institutes

Professor of Medicine, Cellular and Molecular Pharmacology, and Ophthalmology, UC San Francisco

Deputy Director, Innovative Genomics Institute

Bio

Bruce R. Conklin, MD, is a senior investigator at Gladstone Institutes. He is also a professor in the Departments of Medicine, Cellular and Molecular Pharmacology, and Ophthalmology at UC San Francisco, as well as the deputy director of the Innovative Genomics Institute.

Conklin earned a bachelor’s degree in public health from UC Berkeley and completed his medical training at the Case Western Reserve University School of Medicine. During medical school, he spent 2 years as a Howard Hughes Medical Institute Research Scholar in the lab of Nobel laureate Julius Axelrod, PhD, at the National Institute of Mental Health. Conklin completed his residency in internal medicine at Johns Hopkins Hospital, and did his postdoctoral training in molecular pharmacology with Henry Bourne, MD, at UC San Francisco.

How Did You Get Your Start in Science?

“My father was an anthropologist and my mother was a biologist. I studied public policy in college but was inspired by patients to focus on human genetics since medical school.”

Bruce Conklin, MD

Honors and Awards

2016 Case Western Reserve School of Medicine Distinguished Alumni Award

2015 Scientific Advisory Board, Allen Institute for Cell Science

2015 Scientific Advisory Board, Chinese University of Hong Kong, Biological Sciences

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, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health

1988 Harry Resnick Award, Case Western Reserve School of Medicine

Publications

More Publications

Contact

Bruce Conklin

415.734.2712


Lab Members

Alisha Birk
Research Associate I
Matthew Carter
Research Associate I
Claire Clelland, MD, PhD
Visiting Postdoc
Tessa Dewell
Research Associate I
Carissa Feliciano
Research Associate I
John Forsayeth, PhD
Visiting Scientist
Aurelie Fuentes
Student Intern
Hana Ghanim
Research Associate I
Katie Gjoni
Research Associate I
Georgia Gregory
Visiting Researcher
Luke Judge, MD, PhD
Visiting Scientist
Lazaros Lataniotis, PhD
Postdoctoral Scholar
Mohammadali Mandegar
Visiting Scientist
Mo Mandegar, DPhil
Visiting Scientist
Santiago Molina
Visiting Researcher
Juan Perez-Bermejo, PhD
Postdoctoral Scholar
Gokul Ramadoss
Graduate Student
Aradhana Sachdev
Research Associate I
Laney Van Riper
Student Intern (Paid)
Hannah Watry
Visiting Researcher
Beeke Wienert, PhD
Postdoctoral Scholar