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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, PhD
    St. Giles Foundation Professor

Contact

Host

Host Organization

Admission

Free

Tag

Lectures and Seminars

Event RSVP

Dec 201917Tuesday
11:30 AM12:30 PM
The Anlyan CenterN107, TAC Auditorium300 Cedar StreetNew Haven, CT