Genetics Department Seminar Series: "From Base Pairs to Bedside: Antisense Modulation of Splicing or NMD"

I will describe the use of antisense oligonucleotides designed to exploit features of RNA-processing mechanisms so as to alter these reactions for therapeutic purposes. Examples will include: Spinraza, an antisense splicing modulator and the first FDA-approved drug for spinal muscular atrophy; and a method for targeted inhibition of nonsense-mediated mRNA decay, including its potential application to cystic fibrosis.