Science Sundays: Shooting the messenger: 'Antisense' therapies for rare diseases
Schedule
Sun Oct 13 2024 at 03:00 pm to 05:00 pm
UTC-04:00Location
Fawcett Conference Center | Columbus, OH
Join us for the 2nd Annual Schoenberg Lectureship presented by the Center for RNA Biology in partnership with Science Sundays.Ongoing basic research to understand how cells decode the information present in our genes has recently made it possible to develop new types of drugs. We will discuss one of the key steps in this genetic decoding, namely how messenger RNA (mRNA) undergoes splicing. Splicing edits each mRNA to remove non-coding segments, so that the information it carries can be used to make a protein. We will also discuss how mRNA splicing can be precisely manipulated with designer drugs called antisense oligonucleotides (ASOs), for example to correct defective splicing in the context of disease. One notable example we will discuss is an ASO drug that is being used to prevent or treat a deadly neurodegenerative disease.
This event is free and open to the public. RSVPs are requested, but not required to attend. Seating will be on a first come, first served basis.
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Adrian Krainer is the St. Giles Foundation Professor at Cold Spring Harbor Laboratory in Long Island, NY. His lab studies how messenger RNA is naturally produced in human cells, focusing on a crucial editing step called RNA splicing and aiming to understand its mechanism, regulation, and dysfunction in disease. In addition, his lab is engaged in the development of “antisense” drugs that target RNA splicing in the context of various genetic diseases and cancers. Together with Ionis Pharmaceuticals and Biogen, Dr. Krainer’s lab developed an antisense drug called nusinersen (Spinraza), which was approved by the FDA in 2016 as the first treatment for spinal muscular atrophy, a neurodegenerative disease that was the leading genetic cause of infant mortality. His awards and accolades include election to the National Academy of Sciences, the National Academy of Medicine, the National Academy of Inventors, and the American Academy of Arts & Sciences; the Life Sciences Breakthrough Prize, the Lifetime Achievement Award of the RNA Society, the International Prize for Translational Neuroscience, the Speiser Award in Pharmaceutical Sciences, the Takeda Pharmaceuticals Innovators in Science Senior Scientist Award in Rare Diseases, the Ross Prize in Molecular Medicine, the Wolf Prize in Medicine, the Gabbay Award in Biotechnology & Medicine, the Watanabe Prize in Translational Research, and an honorary doctorate from Tel Aviv University. At CSHL, Dr. Krainer has mentored 23 doctoral students and 38 postdoctoral fellows from 22 countries. He served as President of the RNA Society in 2014, and currently serves on the advisory boards of several scientific centers and networks, non-profit foundations, and biotechnology companies in the U.S., Europe, and Latin America.
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Science Sundays is a free lecture series open to the public that provides a wide range of current and emerging topics and issues in science that touch our everyday lives. Speakers are experts in their fields from on campus and around the world with experience in making their topics interesting and accessible for audiences of all ages, with or without a science background.
Each lecture is followed by a free, informal reception from 4-5 p.m.
If you have questions about accessibility or wish to request an accommodation for a disability please contact John Beacom ([email protected]; 614-247-8102). With advance notice of two weeks, we can generally provide seamless access.
Where is it happening?
Fawcett Conference Center, Oxley's Restaurant, 2400 Olentangy River Rd, Columbus, OH 43210, United States,Columbus, Ohio
