Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines

• Spencer C. Wei ,

  Roles Conceptualization, Supervision, Writing – original draft, Writing – review & editing

  * E-mail: spencer.wei@gmail.com

  ¶☯ authors contributed equally

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  https://orcid.org/0000-0001-9673-9683

  ⨯
• Aaron J. Cantor ,

  Roles Formal analysis, Methodology, Supervision, Visualization, Writing – original draft, Writing – review & editing

  ¶☯ authors contributed equally

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  https://orcid.org/0000-0002-2158-6253

  ⨯
• Jack Walleshauser ,

  Roles Investigation, Methodology, Supervision, Writing – original draft

  ¶☯ authors contributed equally

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Rina Mepani,

  Roles Conceptualization, Investigation, Methodology, Supervision

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Kory Melton,

  Roles Data curation, Formal analysis, Investigation, Methodology, Software

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Ashil Bans,

  Roles Investigation, Methodology

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Prachi Khekare,

  Roles Formal analysis, Investigation, Methodology, Software

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Suhani Gupta,

  Roles Investigation, Methodology, Supervision

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Jonathan Wang,

  Roles Investigation, Methodology

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Craig Soares,

  Roles Investigation, Methodology

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  https://orcid.org/0009-0004-8155-4247

  ⨯
• Radwan Kiwan,

  Roles Investigation, Methodology

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Jieun Lee,

  Roles Investigation, Methodology

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Shannon McCawley,

  Roles Investigation, Methodology, Visualization

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Vihasi Jani,

  Roles Investigation, Methodology

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Weng In Leong,

  Roles Investigation, Methodology, Supervision

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Pawan K. Shahi,

  Roles Investigation, Methodology, Visualization, Writing – review & editing

  Affiliations McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, Wisconsin, United States of America, Department of Pediatrics, University of Wisconsin-Madison, Madison, Wisconsin, United States of America

  ⨯
• Jean Chan,

  Roles Investigation, Methodology

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Pierre Boivin,

  Roles Investigation, Methodology, Visualization

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Peter Otoupal,

  Roles Investigation, Methodology, Supervision

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Bikash R. Pattnaik,

  Roles Funding acquisition, Project administration, Supervision, Writing – review & editing

  Affiliations McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, Wisconsin, United States of America, Department of Pediatrics, University of Wisconsin-Madison, Madison, Wisconsin, United States of America, Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, Madison, Wisconsin, United States of America

  ⨯
• David M. Gamm,

  Roles Funding acquisition, Project administration, Supervision, Writing – review & editing

  Affiliations McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, Wisconsin, United States of America, Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, Madison, Wisconsin, United States of America, Waisman Center, University of Wisconsin-Madison, Madison, Wisconsin, United States of America

  ⨯
• Krishanu Saha,

  Roles Funding acquisition, Supervision, Writing – review & editing

  Affiliations McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, Wisconsin, United States of America, Waisman Center, University of Wisconsin-Madison, Madison, Wisconsin, United States of America, Department of Biomedical Engineering, University of Wisconsin-Madison, Madison, Wisconsin, United States of America, Wisconsin Institute of Discovery, University of Wisconsin-Madison, Madison, Wisconsin, United States of America

  https://orcid.org/0000-0003-2837-0858

  ⨯
• Benjamin G. Gowen,

  Roles Supervision, Visualization, Writing – review & editing

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Mary Haak-Frendscho,

  Roles Conceptualization, Funding acquisition, Project administration, Supervision, Writing – review & editing

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• Mary J. Janatpour,

  Roles Conceptualization, Funding acquisition, Project administration, Supervision

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
•  [ ... ],
• Adam P. Silverman

  Roles Conceptualization, Funding acquisition, Project administration, Supervision, Writing – original draft, Writing – review & editing

  Affiliation Spotlight Therapeutics, Hayward, CA, USA

  ⨯
• [ view all ]
• [ view less ]