Work

Bioreducible Gene Delivery Platform that Promotes Intracellular Payload Release and Widespread Brain Dispersion

Year: 2023

Type: article

Abstract: We here introduce a novel bioreducible polymer-based gene delivery platform enabling widespread transgene expression in multiple brain regions with therapeutic relevance following intracranial convection-enhanced delivery. Our bioreducible nanoparticles provide markedly enhanced gene delivery efficacy in vitro and in vivo compared to nonbiodegradable nanoparticles primarily due to the ability to release gene payloads preferentially inside cells. Remarkably, our platform exhibits competitive gene delivery efficacy in a neuron-rich brain region compared to a viral vector under previous and current clinical investigations with demonstrated positive outcomes. Thus, our platform may serve as an attractive alternative for the intracranial gene therapy of neurological disorders.

Source: ACS Biomaterials Science & Engineering

DOI: 10.1021/acsbiomaterials.3c00799

Authors: Divya Rao, Gijung Kwak, Heng Wang, Charles G. Eberhart, Justin Hanes, Jung Soo Suk

Institution: Johns Hopkins University

Language: English

Topic: RNA Interference and Gene Delivery

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: green

Funders: National Institutes of Health, National Eye Institute, National Institute of Neurological Disorders and Stroke