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  • Welcome to the CRISPR Vision Program! Funded by the National Institutes of Health (NIH), we're innovating non-viral gene editing therapies for inherited retinal disorders. Our work aims to bring the groundbreaking potential of CRISPR technology to those affected by these diseases.

     

    About Our Program

    We're pushing the frontiers of gene editing technology. Our focus lies in overcoming the limitations of existing viral delivery systems and introducing novel, non-viral treatments for inherited eye diseases, particularly those affecting the retinal pigment epithelium.

     

    Our Innovative Approach

    Our novel non-viral delivery systems — synthetic nanoparticles and Target Active Gene Editors (TAGE) — sit at the heart of our mission. These promising technologies aim to target the retina efficiently and treat rare conditions like Best Disease and Leber Congenital Amaurosis.

     

    The Impact

    We're pushing the boundaries of gene editing by developing treatments for inherited retinal disorders. Our work could pave the way for treating numerous inherited retinal disorders, marking a significant stride in the fight against genetic eye diseases.

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    About the Somatic Cell Genome Editing (SCGE) Consortium

    As a part of the SCGE Consortium, we are working in a community dedicated to developing safe and effective gene editing methods to treat genetic diseases in somatic cells. Click here for more information about the SCGE NIH Working Group and the Common Fund.

  • Team

    Over 30 members within the program. The leadership team consists of:

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    Kris Saha

    Program Lead, UW-Madison

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    David Gamm

    Project Lead, UW-Madison

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    Shaoqin Gong

    Project Lead, UW-Madison

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    Bikash Pattnaik

    Project Lead, UW-Madison

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    Adam Silverman

    Project Lead, Spotlight Therapeutics

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    William Murphy

    Core Lead, UW-Madison

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    T. Michael Nork

    Core Lead, UW-Madison

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    Jonathan Watts

    Site Lead, UMass Medical School

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    Melissa Skala

    Site Lead, Morgridge Institute

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    Ross Schwartz

    Administrator, UW-Madison

  • Press

    "Innovations in gene-editing treatment in Wisconsin and abroad," The Morning Show, Wisconsin Public Radio, November 28, 2023, Link Listen 

     

    "NIH awards more than $140 million to help accelerate genome editing approaches from the lab to the clinic," National Institutes for Health, Link 

     

    "UW researchers will develop gene editing therapy to treat blindness," University of Wisconsin-Madison, Link

    Associated articles:

    Wisconsin State Journal, Link 

    National Eye Institute Director, Link 

    Foundation for Fighting Blindness, Link

     

    "New gene-editing technique holds potential for treating childhood blindness," University of Wisconsin-Madison, Link

  • Selected Publications

    Kabra, M., Shahi, P. K., Wang, Y., Sinha, D., Spillane, A., Newby, G. A., Saxena, S., Tong, Y., Chang, Y., Abdeen, A. A., Edwards, K. L., Theisen, C. O., Liu, D. R., Gamm, D. M., Gong, S., Saha, K., & Pattnaik, B. R. (2023). Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy. The Journal of Clinical Investigation. Link  Press


    Sinha, D., Steyer, B., Shahi, P. K., Mueller, K. P., Valiauga, R., Edwards, K. L., Bacig, C., Steltzer, S. S., Srinivasan, S., Abdeen, A., Cory, E., Periyasamy, V., Siahpirani, A. F., Stone, E. M., Tucker, B. A., Roy, S., Pattnaik, B. R., Saha, K., & Gamm, D. M. (2020). Human iPSC Modeling Reveals Mutation-Specific Responses to Gene Therapy in a Genotypically Diverse Dominant Maculopathy. The American Journal of Human Genetics, 107(2), 278–292. Link

     

    Saha, K., Sontheimer, E. J., Brooks, P. J., Dwinell, M. R., Gersbach, C. A., Liu, D. R., Murray, S. A., Tsai, S. Q., Wilson, R. C., Anderson, D. G., Asokan, A., Banfield, J. F., Bankiewicz, K. S., Bao, G., Bulte, J. W. M., Bursac, N., Campbell, J. M., Carlson, D. F., Chaikof, E. L., … SCGE Consortium. (2021). The NIH Somatic Cell Genome Editing program. Nature, 592(7853), 195–204. Link

     

    Chen, G., Abdeen, A. A., Wang, Y., Shahi, P. K., Robertson, S., Xie, R., Suzuki, M., Pattnaik, B. R., Saha, K., & Gong, S. (2019). A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing. Nature Nanotechnology, 14(10), 974–980. Link

    Wang, Y., Shahi, P. K., Xie, R., Zhang, H., Abdeen, A. A., Yodsanit, N., Ma, Z., Saha, K., Pattnaik, B. R., & Gong, S. (2020). A pH-responsive silica-metal-organic framework hybrid nanoparticle for the delivery of hydrophilic drugs, nucleic acids, and CRISPR-Cas9 genome-editing machineries. Journal of Controlled Release, 324, 194–203. Link

     

    Samimi, K., Pattnaik, B. R., Capowski, E. E., Saha, K., Gamm, D. M., & Skala, M. C. (2022). In situ autofluorescence lifetime assay of a photoreceptor stimulus response in mouse retina and human retinal organoids. Biomedical Optics Express, 13(6), 3476–3492. Link 

    Wang, Y., Ma, B., Abdeen, A. A., Chen, G., Xie, R., Saha, K., & Gong, S. (2018). Versatile Redox-Responsive Polyplexes for the Delivery of Plasmid DNA, Messenger RNA, and CRISPR-Cas9 Genome-Editing Machinery. ACS Applied Materials & Interfaces. Link 

    Steyer, B., Bu, Q., Cory, E., Jiang, K., Duong, S., Sinha, D., Steltzer, S., Gamm, D., Chang, Q., & Saha, K. (2018). Scarless Genome Editing of Human Pluripotent Stem Cells via Transient Puromycin Selection. Stem Cell Reports, 10(2), 642–654. Link 

     

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    Overview of the genome editing strategy for two rare inherited diseases: Leber congenital amaurosis (top) and Best disease (bottom). Channel proteins in the cell membrane of retinal cells are shown on the left. The gene editing strategy on the right will modify the mutant gene in order to fix the mutations and establish functional channel proteins.

     

    Additional technical information is here.

     

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    Progress and Goals

    Keep up with our progress. We're committed to developing our non-viral gene editing therapies, creating a platform to tackle various rare eye diseases, and providing clarity on the development path for new gene-editing therapies. Stay tuned for updates on our journey to turn the promise of CRISPR technology into a reality.