Oligonucleotide-based gene therapy 

Optimizing the chemical architecture of siRNA improves its therapeutic potential for neurodegenerative disorders.

Small interfering RNAs (siRNAs) are a new class of oligonucleotide therapeutics that degrade disease-causing mRNA. However, restrictions imposed by the blood-brain barrier and short circulation times limit the therapeutic potential of siRNAs for brain disorders. In this webinar, Julia Alterman will discuss an innovative approach to achieve potent and long-term siRNA activity in the brain using a novel chemical scaffold that enables widespread siRNA brain delivery and gene silencing.

Topics to be covered

  • A novel siRNA chemical scaffold for widespread brain delivery
  • Potent and long-term gene silencing in the mouse and nonhuman primate brain

Wednesday, October 11th, 2023 | 2:00 PM - 3:00 PM Eastern Time
This webinar will be available to view live and on demand.


Julia Alterman, PhD

Julia Alterman, PhD

Assistant Professor
University of Massachusetts Chan Medical School

Supported by