Human Health

program Leadership

Alex Marson, M.D., Ph.D.
Director, Human Health

Fyodor Urnov, Ph.D.
Director, Technology & Translation

The IGI is working to make CRISPR the standard of care for all genetic diseases.

Mutations in single genes cause more than 5,000 human diseases. Scientists have known the cause of many genetic disorders for years, but until now there has been no way to treat them. The IGI aims to use CRISPR-based treatments to correct disease-causing mutations in cells, animal models, and eventually human patients.

Hand holding slides with sections of tissue. © Noah Berger 2016

Key Goals

Discovery: Use CRISPR screens and machine learning to expand our understanding of the genetic basis of complex diseases and identify new therapeutic targets

Development: Streamline the creation of new CRISPR-based therapies and diagnostics for neglected diseases and areas of high unmet need

Translation: Accelerate the process of bringing accessible and scalable CRISPR cures from the lab bench to clinic

Center for Translational Genomics

The IGI Center for Translational Genomics (CTG) is a first-of-its-kind innovation hub for gene-editing research and development of targeted treatments for intractable genetic diseases. The CTG's aim is to accelerate the timeline from a diagnosis to a cure, and eventually provide customized treatments to individuals living with these conditions.

Flagship Projects

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Sickle Cell Initiative

We are advancing the first not-for-profit clinical trial of a genomic medicine for sickle cell disease.

IGI investigators are working with a consortium of researchers at UC Berkeley, UCSF, and UCLA to begin a clinical trial in 2022, using CRISPR technology developed at the IGI to directly correct the mutation that causes sickle cell disease Our holistic approach to sickle cell disease includes partnering with the sickle cell community, public education, and finding new models to make therapies accessible to those who need it.

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Developing Next-Generation Diagnostics

We are creating CRISPR-based diagnostics for unmet needs.

IGI researchers and the IGI Clinical Laboratory are working on two types of diagnostics: rapid CRISPR-based diagnostic tests for infectious diseases, and companion diagnostics to assess the safety and efficacy of new genome-editing therapies in clinical trials at a molecular level.

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Treating a Genetic Autoimmune Condition

We are advancing a clinical trial tailored to an individual's unique genetic make-up.

CRISPR genome editing opens the door for individualized medicine, allowing the creation of genomic medicines that can treat just one family, or just one person. Our researchers are working with the FDA to move forward with a treatment for an individual with a previously unknown severe autoimmune condition, paving the way for future therapies for rare and neglected diseases.

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