한빛사논문
- 조회497
Seungkuk Ahn 1, Oliver Siontas 1, Janis Koester 2, Jacek Krol 2, Sascha Fauser 2, Daniel J Müller 1
1Eidgenössische Technische Hochschule (ETH) Zürich, Department of Biosystems Science and Engineering, Basel, 4056, Switzerland.
2F. Hoffmann-La Roche Ltd, Roche Pharma Research and Early Development, Department of Ophthalmology, Basel, 4070, Switzerland.
CORRESPONDING AUTHORS: Sascha Fauser, Daniel J Müller
Abstract
Adeno-associated viruses (AAVs) are intensively explored for gene therapies in general and have found promising applications for treating retina diseases. However, controlling the specificity (tropism) and delivery of AAVs to selected layers, cell types, and areas of the retina is a major challenge to further develop retinal gene therapies. Magnetic nanoparticles (MNPs) provide effective delivery platforms to magnetically guide therapeutics to target cells. Yet, how MNPs can deliver AAVs to transfect particular retina layers and cells remains elusive. Here, we demonstrate that MNPs can be used to transport different AAVs through the retina and to modulate the selective transduction of specific retinal layers or photoreceptor cells in ex vivo porcine explants and whole eyes. Thereby, transduction is triggered by bringing the viruses in close proximity to the target cell layer and by controlling their interaction time. We show that this magnetically guided approach to transport AAVs to selected areas and layers of the retina does not require the cell-specific optimization of the AAV tropism. We anticipate that the new approach to control the delivery of AAVs and to selectively transduce cellular systems can be applied to many other tissues or organs to selectively deliver genes of interest.
논문정보
- Research article
- 2024년 06월 (BRIC 등록일 2024-06-12)
- 국외 연구진
- 바이오·의료융합 > 바이오소재
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