TARGETED NANOCARRIERS FOR TARGETED DRUG DELIVERY OF GENE THERAPEUTICS

摘要

The present invention relates to targeted nanocarriers - also termed nanomedicines - and methods of preferentially, or actively, targeting and delivering a tool for gene transfer or genome editing (i.e., a plasmid or a restriction enzyme - such as a zinc finger nuclease, a CRISPR/Cas system, or a TALEN) or a tool for gene silencing or post-transcriptional regulation of gene expression (i.e., a microRNA, a siRNA, a mRNA, an antisense oligonucleotide, or a sense oligonucleotide) to a range of mammalian cell species. Cell- specific targeting is achieved by using nanocarriers featuring suitable targeting anchors having a targeting moiety that can be a carbohydrate, an antibody or an antibody fragment, a non-antibody protein derivative, an aptamer, a lipoprotein or a fragment thereof, a peptidoglycan, a lipopolysaccharide or a fragment thereof, or a CpG DNA. Such targeting anchors may or may not include a polymeric spacer like polyethylene glycol. The nanomedicines shall allow to therapeutically address a range of mammalian disease entities via various application routes. These indications include malignant diseases, autoimmune diseases, inherited disorders, metabolic disorders, or infectious diseases.