Friday 4th November — at 12.00 pm — Amphi J.Monod (Bât.66)
Invited Speaker : Michelle L. Hastings
From Associate Professor , Chicago Medical School
Rosalind Franklin University of Medicine and Science
Department of Cell Biology and Anatomy
Hosted by Christian Muchardt
Abstract : Therapeutics designed to target RNA offer powerful new strategies for the treatment of disease. Antisense oligonucleotide (ASO) technology is one such RNA-targeting tool that is being used to directly modulate gene expression for therapeutic benefit. We work with a type of ASO that is chemically modified to bind with high-affinity to pre-mRNA and create a steric block to splicing. These so-called splice-switching oligonucleotides (SSOs) are effective at correcting defective splicing associated with disease. In this talk, I will discuss the utility of SSOs as drugs and highlight some of our studies to design and develop therapeutic SSOs for hearing impairment, vestibular dysfunction and vision loss in Usher syndrome and neurodegeneration in Alzheimer’s disease.