Defining the Rules for Designing Fully Chemically Modified siRNAs to Treat Genetically Linked Central Nervous System Disorders
Sarah Davis | Khvorova Lab | F31 Award
Small interfering RNA (siRNA) therapeutics are a promising class of drugs for the treatment of genetic disorders. Chemical modification of siRNA is necessary for delivery to the central nervous system, but may hinder the efficacy of some siRNAs. This project will define the relationships between siRNA sequence, chemical modification patterns, and efficacy to streamline the development of clinically-relevant siRNAs capable of treating genetically-defined neurological disorders.
