| abstract |
A ribonucleoprotein (RNP) delivery system comprising (a) a cationic lipid; (b) a phospholipid; (c) a peptide of the structure A-B-C in which A is a polycationic nucleic acid-binding component, B is a spacer element comprising two or more amino acid residues, and C is a cell surface receptor binding component; and (d) a ribonucleoprotein (RNP) complex comprising a CRISPR-associated protein and guide RNA, pharmaceutical compositions comprising such a ribonucleoprotein (RNP) delivery system, and methods for the treatment or prophylaxis of a condition caused in a human or in a non-human animal by a defect and/or a deficiency in a gene, or for RNA therapy, or for the treatment of a cancer, which comprises administering the RNP delivery system or pharmaceutical composition to the human or to the non-human animal. |