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Publication of the Month | October 2021

    Home Publication of the Month Publication of the Month | October 2021

    Publication of the Month | October 2021

    By The McGowan Institute For Regenerative Medicine | Publication of the Month, Publication of the Month 2021 | Comments are Closed | 28 October, 2021 | 0

    Author: Bilge E Öztürk, Molly E Johnson, Michael Kleyman, Serhan Turunç, Jing He, Sara Jabalameli, Zhouhuan Xi, Meike Visel, Valérie L Dufour, Simone Iwabe, Felipe Pompeo Marinho, Gustavo D Aguirre, José-Alain Sahel, David V Schaffer, Andreas R Pfenning, John G Flannery, William A Beltran, William R Stauffer, Leah C Byrne

    Title: scAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution

    Summary: Background: Adeno-associated virus (AAV)-mediated gene therapies are rapidly advancing to the clinic, and AAV engineering has resulted in vectors with increased ability to deliver therapeutic genes. Although the choice of vector is critical, quantitative comparison of AAVs, especially in large animals, remains challenging.

    Methods: Here, we developed an efficient single-cell AAV engineering pipeline (scAAVengr) to simultaneously quantify and rank efficiency of competing AAV vectors across all cell types in the same animal.

    Results: To demonstrate proof-of-concept for the scAAVengr workflow, we quantified – with cell-type resolution – the abilities of naturally occurring and newly engineered AAVs to mediate gene expression in primate retina following intravitreal injection. A top performing variant identified using this pipeline, K912, was used to deliver SaCas9 and edit the rhodopsin gene in macaque retina, resulting in editing efficiency similar to infection rates detected by the scAAVengr workflow. scAAVengr was then used to identify top-performing AAV variants in mouse brain, heart and liver following systemic injection.

    Conclusions: These results validate scAAVengr as a powerful method for development of AAV vectors.

    Source: eLife 2021;10:e64175 DOI: 10.7554/eLife.64175

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    • Home
    • Our People
      • Faculty/Staff Bios
      • Core Faculty Publications
      • Administrative Resources
    • Our Technologies
    • About Us
      • Welcome
      • Video
      • Mission Statement
      • What Is Regenerative Medicine?
      • Executive Committee
      • Contact Us
      • Clinical Site
    • Our Research
      • Focus Areas
        • Tissue Engineering and Biomaterials
        • Cellular Therapies
        • Medical Devices and Artificial Organs
        • Clinical Translation
      • Matrix
      • Centers
      • Laboratories
      • Clinical Trials
      • Initiatives
    • Media
      • Current News
      • News Archive
      • Video
      • Podcasts
      • Newsletter
      • Grant of the Month
      • Publication of the Month
      • Media Contact
      • Video Links
    • Professional Development
      • Seminar Series
      • Special Events
      • Student Interest Groups
      • CATER
      • Post-Doctoral Opportunities
      • Career Opportunities
      • Wiegand Summer Internship
      • Admissions
      • Summer School
      • 2022 Scientific Retreat
    Regenerative Medicine at the McGowan Institute