MUSCLE CELL-TARGETING NANOPARTICLES FOR VACCINATION AND NUCLEIC ACID DELIVERY, AND METHODS OF PRODUCTION AND USE THEREOF

摘要

Described herein are conjugates (e.g., nanoparticles), compositions (e.g., nanoparticle-based compositions), kits, methods and platforms for muscle-specific targeting of vaccines and gene therapies. The nanoparticles are composed of charged polymers or charged particles (e.g., polyamidoamide dendrimers) surface-modified with a muscle cell ligand (a targeting moiety) and an anionic enhancer (e.g. a phospholipid) resulting in a multinucleotide-carrying conjugate for introducing vaccines or genes into skeletal muscle cells of a subject (e.g., human and other mammals). The novel compositions described herein find use in muscle-targeted vaccination and gene therapy delivery (e.g., delivery of gene-based vaccines) for the prevention and treatment of disease (e.g., skeletal muscle atrophy, cancer, autoimmune disease, infection).