A 3D illustration of a human brain, an organ that scientists can target with oligonucleotide gene therapy to treat neurological diseases.
Webinars

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.


Speaker

Julia Alterman, PhD

Julia Alterman, PhD

Assistant Professor
University of Massachusetts Chan Medical School

Top Image Credit:

Credit: iStock/ Jolygon