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.
Speaker
Cold Spring Harbor Laboratory
Adrian Krainer, PhDSt. Giles Foundation Professor
Contact
Host
Host Organization
Admission
Free
Tag
Lectures and Seminars