Development and characterization of a first-in-class adjustable-dose gene therapy system.

ABSTRACT:

Introduction

Despite significant potential, gene therapy has been relegated to the treatment of rare diseases, due in part to an inability to adjust dosage following initial administration. Other significant constraints include cost, specificity, antigenicity, and systemic toxicity of current generation technologies. To overcome these challenges, we developed a first-in-class adjustable-dose gene therapy system, with optimized biocompatibility, localization, durability, and cost.

Methods

A lipid nanoparticle (LNP) delivery system was developed and characterized by dynamic light scattering for size, zeta potential, and polydispersity. Cytocompatibility and transfection efficiency were optimized in vitro using primary human adipocytes and preadipocytes. Durability, immunogenic

SUBMITTER: Goraltchouk A 

PROVIDER: S-EPMC12831582 | biostudies-literature | 2024 Aug

REPOSITORIES: biostudies-literature