Genome Editing Research Program – UCI Health Affairs

Our Vision: Cure Genetic Disorders

Gene editing holds immense promise for the future of medicine, offering hope to millions affected by genetic disorders.

This groundbreaking technology has the potential to precisely correct genetic mutations at their source, addressing the root cause of conditions that were once deemed untreatable. From inherited vision disorders to more widespread genetic conditions such as cystic fibrosis, sickle cell anemia and muscular dystrophy, gene editing represents a revolutionary approach to treatment. By restoring normal function at the cellular level, gene editing not only alleviates symptoms but also aims to provide lasting cures, transforming the lives of patients and their families and ushering in a new era of medical possibilities.

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, and new expert recruits, will collaborate in custom-designed labs to translate discoveries into cures.

Interdisciplinary teams will work side-by-side to model and understand inherited disorders, and then develop and test therapies to benefit patients.

Future leaders will be trained by the best in leading-edge science and medicine.

Indoor and outdoor gathering spaces for events, speakers and presentations will foster engagement and dialogue to speed and share discoveries.

Innovating Genetic Therapies

Introduced just a decade ago, gene editing has swiftly emerged as one of the most promising frontiers in innovative approaches to restoring 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.

Together, we can pave the way for a future where genetic disorders are not just managed but cured. Your support can make a significant difference in the lives of countless individuals and families affected by inherited diseases and conditions.

Our Competitive Advantage

The strength of the Genome Editing Research Program is its unique synergy of multifaceted research laboratories, each specializing in complementary aspects of functionality and disease. These labs cover a broad spectrum of disciplines, including structural biology, genetics, physiology and pharmacology.

This interdisciplinary approach fosters a rich environment of collaboration, enabling dedicated investigators to model and understand a wide array of genetic disorders comprehensively. Our commitment to the One Health model, a hallmark of UC Irvine Health Affairs, exemplifies this collaborative spirit by uniting various health disciplines to amplify their strengths.

By working together within this integrative framework, our experts are able to develop and test novel therapies with the ultimate goal of delivering tangible benefits to patients. This approach not only accelerates the pace of discovery but also ensures that the solutions developed are both holistic and highly effective, addressing the complexities of genetic diseases from multiple angles.

Key Research Areas in Gene Editing: Transformative Projects in Human Health

Through collaborative efforts, we are creating a unique research environment conducive to discovering breakthroughs in the treatment of ocular diseases. Projects focusing on conditions such as retinitis pigmentosa and macular degeneration are making significant strides in addressing inherited eye diseases. By using CRISPR technology to correct genetic mutations, Krzysztof Palczewski, PhD, and his team aim to restore vision and prevent further degeneration in patients.

Glaucoma

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 leading-edge research with compassionate patient care, ensures our work goes beyond laboratory experiments to make a significant difference in patients’ lives.

Retina

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, restoring vision from the retina to the visual cortex and providing 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.

Current Team

We have assembled, and will seek to expand, an exceptional group of scientists with a keen understanding of genome editing to optimize research collaborations. Faculty leadership includes:

Krzysztof Palczewski, PhD

Founding Director of the Genome Editing Research Program; Director of the Center for Translational Vision Research

Rui Chen, PhD

Professor, Ophthalmology, Biomedical Engineering

Audrey Lapinaite, PhD

Associate Professor, Ophthalmology, Biomedical Engineering

Chang Liu, PhD

Professor, Biomedical Engineering, Molecular Biology and Biochemistry

Gregory Weiss, PhD

Professor, Chemistry, Molecular Biology and Biochemistry, Pharmaceutical Sciences

Gulab Zode, PhD

Professor, Ophthalmology, Physiology & Biophysics

Join Us in Making an Impact

We are seeking philanthropic partners for the Genome Editing Research Program in the Falling Leaves Foundation Medical Innovation Building to provide funding that will drive faculty recruitments and 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 UC Irvine’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 UC Irvine’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.