In Vivo Cytosolic Delivery of Biomolecules into Neurons for Super-Resolution Imaging and Genome Modification.

ABSTRACT: Efficient delivery of biomolecules into neurons has significant impacts on therapeutic applications in the central nervous system (CNS) and fundamental neuroscience research. Existing viral and non-viral delivery methods often suffer from inefficient intracellular access due to the endocytic pathway. Here, a neuron-targeting and direct cytosolic delivery platform is discovered by using a 15-amino-acid peptide, termed the N1 peptide, which enables neuron-specific targeting and cytosolic delivery of functional biomolecules. The N1 peptide initially binds hyaluronan in the extracellular matrix and subsequently passes the membrane of neurons without being trapped into endosome. This mechanism facilitates the efficient delivery of cell-impermeable and photo-stable fluorescent dye for super-reso

SUBMITTER: Ge X 

PROVIDER: S-EPMC12224921 | biostudies-literature | 2025 Apr

REPOSITORIES: biostudies-literature