Nucleic Acid Delivery to the Liver for Hemophilia and Lysosomal Storage Disease Treatment (20150339, Dr. Reineke)

Polymerized GNA Ligands Deliver Nucleic Acids to the Liver

Polymeric materials containing polymerized GNA ligands deliver nucleic acids to the liver with low cytotoxicity. A series of cationic diblock glycopolymers derived from N-acetyl-D-galactosamine (GNA) target asialoglycoprotein (ASGP) receptors on hepatocytes in the liver. These easily produced polymers bind to nucleic acids to form nanoparticles that cells efficiently take up. This novel method of nucleic acid delivery to the liver has many therapeutic applications, especially for hemophilia and several lysosomal storage diseases.

Lower Cytotoxicity in Liver-Targeted Delivery

Existing technologies for liver-targeted delivery use either viral or lipid-based materials, which can be toxic. Using these polymerized GNA ligands demonstrates lower cytotoxicity than the existing viral or lipid-based technologies. Furthermore, this polymerized form of GNA ligands is novel: until now, GNA ligands have only been used in mono, bi, or triantennary structures.

BENEFITS AND FEATURES:

• Delivers nucleic acids to the liver
• Lower cytotoxicity compared to existing viral or lipid-based materials
• Easily produced
• Novel polymerized form of GNA ligands
• Targets ASGP receptor-specific cell types

APPLICATIONS:

• Nucleic acid delivery to liver in vivo or ASGP receptor-specific cell types
• Treatment of hemophilia and lysosomal storage diseases

Researchers