About The Author

Anastasia Khvorova currently works at the RNA Therapeutics Institute (RTI), University of Massachusetts Medical School. Her Laboratory focuses on medicinal chemistry of therapeutic RNAs for productive delivery to extrahepatic tissues.

Disrupting The Brain Keeper To Allow Silencing Of Deleterious Genes In The Nervous System

Therapeutic oligonucleotides, such as small interfering RNAs (siRNA), hold great promise for the treatment of genetically-defined disorders, including diseases of the central nervous system (CNS). These oligonucleotide-based drugs effectively and potently modulate gene expression of toxic RNAs and proteins, potentially blocking or reversing disease progression. Despite the significant enthusiasm of the scientific community after their