Focused ultrasound as a novel strategy for noninvasive gene delivery to retinal Müller glia
Yacine Touahri, Rajiv Dixit, Rikke Hahn Kofoed, Kristina Miloska, EunJee Park, Reza Raeisossadati, Kelly Markham-Coultes, Luke Ajay David, Hibo Rijal, Jiayi Zhao, Madelaine Lynch, Kullervo Hynynen, Isabelle Aubert, Carol Schuurmans
Abstract
: Retinal permeabilisation via FUS in the presence of microbubbles is effective for delivering AAV2/8 across the inner blood-retinal-barrier, targeting Müller glia, which is less invasive than intravitreal injections that bypass the inner limiting membrane. However, implementing FUS in the clinic will require a comprehensive consideration of any off-target tropism of the AAV in peripheral organs, combined ideally, with the development of Müller glia-specific promoters.
Topics & Concepts
Gene deliverymCherryMuller gliaRetinalGreen fluorescent proteinRetinaBiologyTransduction (biophysics)Reporter geneEx vivoCell biologyMicrobubblesGenetic enhancementViral vectorIn vivoStem cellProgenitor cellRecombinant DNANeuroscienceGene expressionMedicineGeneUltrasoundBiochemistryGeneticsRadiologyRetinal Development and DisordersVirus-based gene therapy researchPhotoacoustic and Ultrasonic Imaging