Focused ultrasound as a novel strategy for noninvasive gene delivery to retinal M ü ller glia

Conclusions: 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.
Source: Theranostics - Category: Molecular Biology Authors: Tags: Research Paper Source Type: research