Systemic administration of small-molecule therapeutics can lead to off-target toxicity and low doses at sites requiring treatment. To mitigate these issues while improving pharmacokinetics and bioavailability, scientists have turned to nanomaterials that house drug cocktails and have longer blood circulation half-lives. However, most nanomaterials lack active targeting agents that direct the drug delivery vehicle to the site of diseased tissue, and even fewer can specifically target the kidney since they are too large. We propose to develop novel matrix protein-drug conjugates that bear new biocompatible polymers. These will simultaneously target kidney glomeruli, function as imaging agents, and locally deliver combinations of therapeutics. This research could lead to new approaches to treating pediatric kidney disease.
· Synthesize and test new protein-polymer conjugates as renal targeting delivery in proof of concept studies.
· Test the safety and efficacy of therapeutic drugs delivered by novel protein-polymer vehicles in mouse models of pediatric kidney diseases.
Potential impact on child health
The innovative approach of this research will result in a promising new platform for personalized treatment of acute and chronic pediatric kidney diseases.