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Nanomedicine and Nanoscale Delivery (Focus Group – NND)

Investigating CNS delivery of ASOs formulated on DNA-based virus-like particles

Sarah Al-Abdullatif – Postdoctoral Fellow, Massachusetts Institute of Technology; Mark Bathe – Professor, Massachusetts Institute of Technology; Kha Uyen Dam – Graduate Student, UMass Chan Medical School; Grant Knappe – Postdoctoral Associate, Massachusetts Institute of Technology; Rachael Sirianni – Professor, UMass Chan Medical School

    Poster Presenter(s)

  • DL

    Duy An Le

    Graduate Student
    Massachusetts Institute of Technology, United States

Introduction:

Antisense oligonucleotides (ASOs) have emerged as a promising class of genetic medicines aimed at regulating the levels of target RNAs within the cell. When administered in the central nervous system (CNS) via intrathecal administration, ASOs distribute broadly and indiscriminately across cell types, limiting therapeutic precision.1 To investigate alternative delivery methods for ASOs, we engineered DNA-based virus-like particles (D-VLPs). Using the DNA origami technique, enabling control over D-VLP size and geometry, as well as the multimerization of ligands and cargos in a spatially controlled manner.2

Learning Objectives:

  • contrast the biodistribution and efficacy profiles of ASOs delivered in nanoparticulate form and as bolus.
  • discover a novel strategy for delivery, built on the DNA origami technique.
  • identify key challenges in region, and cell-specific delivery of oligonucleotides in the CNS.