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Accelerating CRISPR cures from the lab to the clinic
Worldwide, there are over 6000 genetic diseases, most of which have few treatment options and no cures. Medical research typically targets one disease at a time, focusing on common diseases with large patient populations, and it can take a decade or more to develop each new therapy.
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, housed in the IGI Building on UC Berkeley campus. IGI’s CTG team is working to create a platform to enable new therapies for genetic blood and primary immune disorders, focusing on rare and neglected conditions. We aim to accelerate the timeline from a diagnosis to a cure, working with our clinical partners to provide customized treatments to individuals living with these conditions.
When developing CRISPR-based therapies for a genetic condition, most of the steps between a patient and cure are the same. For blood or primary immune disorders, the patient’s blood stem cells are removed from the body, genetically edited using CRISPR machinery, then returned back to the patient. What is different is which gene is being edited.
The guide RNA – a short sequence that is specific to the gene of interest – tells the CRISPR machinery exactly where to edit DNA. By swapping out the guide RNA, we can target the CRISPR machinery to any gene in the genome. In other words, we can potentially cure any genetic blood disease by changing the sequence of our guide RNA.
Current approaches target one disease at a time. Our approach will create ready-to-deploy treatments for a range of diseases: we are developing an automated system to design and test thousands of guide RNAs for genes involved in inherited bone marrow failure syndromes or primary immunodeficiency. With this pipeline, we will be able to rapidly identify the best place in a disease-causing gene to target with CRISPR machinery. We will annotate all known genes involved in these diseases with optimized guide RNAs, so we will be ready to treat patients when the time comes.
The Interventional Genomics Unit for Therapeutic Innovation (INGENUITI) is an initiative at the Innovative Genomics Institute to study the mechanisms underlying how genomic variants can cause disease and how gene editing tools like CRISPR-Cas9 could be used to treat those diseases. We are actively recruiting participants for this study, which you can read more about here. If you’re interested, you can email ingenuiti@berkeley.edu, and an investigator will get back to you.
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(2023)Foss DV, Muldoon JJ, Nguyen DN, Carr D, Sahu SU, Hunsinger JM, Wyman SK, Krishnappa N, Mendonsa R, Schanzer EV, Shy BR, Vykunta VS, Allain C, Li Z, Marson A, Eyquem J, and Wilson RC. Nature Biomedical Engineering
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(2022)Magis W, DeWitt MA, Wyman SK, Vu JT, Heo S-J, Shao SJ, Hennig F, Romero ZG, Campo-Fernandez B, Said S, McNeill MS, Rettig GR, Sun Y, Wang Y, Behlke MA, Kohn DB, Boffelli D, Walters MC, Corn JE, and Martin DIK. iScience
(2022)To learn about open positions at the IGI and CTG, see our Careers page.
For any inquiries about the CTG’s ongoing work or potential partnerships, please contact Madalyn Lesman at mlesman@berkeley.edu.
