Founding Director: Krzysztof Palczewski, PhD
Our Vision: Cure Genetic Disorders
Introduced just a decade ago, gene editing has swiftly emerged as one of the most promising frontiers in the quest for innovative approaches to restore DNA sequences.
At UC Irvine, our investigators have unlocked the ability to reverse and forestall cell degeneration. Currently, our researchers are tirelessly working to refine a method for directing this therapy to specific cells in need of correction. The pioneering approaches spearheaded by the Genome Editing Research Program, once realized, hold the potential to revolutionize not only the treatment of genetic disorders affecting vision but also a broader spectrum of inherited conditions.
The program has been designated as one of the 12 high-impact research programs to be housed in the Falling Leaves Foundation Medical Innovation Building.
In This New Facility
Current world-renowned scientists and clinicians, alongside newly recruited experts, will collaborate in custom-designed labs to translate discoveries into cures.
Interdisciplinary teams will collaborate closely to model and comprehend inherited disorders, then develop and test therapies for the benefit of patients.
Future leaders will receive training from the forefront of science and medicine.
Indoor and outdoor gathering spaces will facilitate events, speakers and presentations, fostering engagement and dialogue to expedite and share discoveries.
Our Competitive Advantage
Empowered by the distinctive One Health approach of UCI Health Affairs that transcends disciplinary boundaries, the Genome Editing Research Program comprises faculty and staff from the Susan & Henry Samueli College of Health Sciences (encompassing schools of medicine, nursing, pharmacy and pharmaceutical sciences, population and public health) and UCI Health, our regional healthcare delivery system which includes the trailblazing Susan Samueli Integrative Health Institute. By nurturing collaborative efforts among brilliant minds, our teams can surpass the limitations of working in isolation. This collaborative approach enables us to push the boundaries of innovation and make fundamental discoveries in the field of gene therapy.
The strength of the Genome Editing Research Program lies in a critical mass of multifaceted research laboratories, each focusing on complementary aspects of function and disease, ranging from structural biology and genetics to physiology and pharmacology. Dedicated investigators collaborate to model and understand disorders, subsequently developing and testing therapies to benefit patients.
Retina Research
In the realm of retinal research, our program is addressing the urgent need for effective treatments for inherited retinal diseases, which currently have limited therapeutic options. Our innovative use of base and prime editing has led to the correction of mutations responsible for conditions such as Leber Congenital Amaurosis and Retinitis Pigmentosa, not only restoring vision from the retina to the visual cortex but also offering a protective shield for photoreceptors against degeneration. These groundbreaking advancements have been published in prestigious journals such as Cell, Nature Biotechnology, Nature Biomedical Engineering, Nature Communications and PNAS. We are dedicated to refining our genome engineering techniques and developing new delivery methods to ensure our genome editing is safer, more effective and more precise. This effort demonstrates our steadfast commitment to advancing the field of genetic therapy.
Glaucoma Research
Glaucoma is the second leading cause of irreversible blindness worldwide. We are paving the way to innovative treatments through studies that successfully target the mutant MYOC gene using the CRISPR-Cas9 system, significantly reducing mutant myocilin levels in the trabecular meshwork and preventing the onset of glaucoma in models. This research is instrumental in developing targeted delivery systems for various mutations associated with glaucoma. Our holistic approach, combining cutting-edge research with compassionate patient care, ensures our work goes beyond laboratory experiments to make a significant difference in patients’ lives. Through collaborative efforts, we are creating a unique research environment conducive to discovering breakthroughs in the treatment of ocular diseases.
Join Us in Making an Impact
We invite philanthropic partners to collaborate with us in establishing a new home for the UCI Genome Editing Research Program within the Falling Leaves Foundation Medical Innovation Building, and in providing essential support for our programmatic funding to drive transformative discoveries.
Named Spaces Within the Falling Leaves Foundation Medical Innovation Building
The architectural design of the Falling Leaves Foundation Medical Innovation Building is tailored to facilitate vibrant collaborations that translate inspiration into tangible results. You have the exclusive opportunity to name the new headquarters for the Genome Editing Research Program and custom-designed laboratories and gathering spaces. As a philanthropic partner, your contribution will ensure that our scientists have the necessary space, resources and facilities to advance their life-changing work.
Endowed Research Grants
Research endowments play a pivotal role in sustaining our investigators, who are at the forefront of shaping the future of science and healthcare. External grants cover only a fraction of associated expenses, underscoring the importance of endowed funding in supporting innovative discoveries and pioneering approaches. Endowed grants bear special significance, offering the flexibility to pursue scientific avenues that lead to breakthroughs. Your generous support will enable this vital work to be carried out under the auspices of our philanthropic partners. Additionally, named endowed scholarships will further enrich the training of future generations of leading investigators and healthcare providers in perpetuity.
Endowed Chairs
By partnering with us as a philanthropic sponsor to name an endowed chair, you will establish a lasting connection between your legacy and UCI’s esteemed experts. Ultimately, advancements in biomedical research will drive the development of innovative therapies. Philanthropic contributions towards endowed chairs, research and scholarships ensure sustained support, facilitating the recruitment and retention of top-tier faculty who are instrumental in maintaining UCI’s status as a world-class center of excellence in emerging therapies. Endowed chairs are particularly impactful as they establish a perpetual annual funding stream, empowering the chair-holder to focus their efforts on groundbreaking research initiatives.
Partner With Us
Please join us as we strive to turn our vision for the Genome Editing Research Program into reality. To learn more about exclusive naming opportunities for this transformative initiative, as well as associated spaces within the medical innovation building, please contact:
Caroline Pereira, MBA
Associate Vice Chancellor of Health Advancement
caroline.pereira@uci.edu | 949-824-5577
