Originally Aired: August 17, 2023
Time: 8:00 am PT, 11:00 am ET, 17:00 CET
Targeting mRNA expression presents a promising approach in the treatment of haploinsufficiency disorders, offering a new avenue for therapeutic intervention. While gene expression changes are commonly attributed to programmed transcriptional variability, it is noteworthy that mRNA expression undergoes extensive regulation during translation, a lesser-known aspect. The rate of mRNA translation plays a crucial role in post-transcriptional regulation. Within cells, translational regulation is achieved through specific sequence and/or structural elements that recruit factors with positive or negative effects on mRNA function. Each transcript exhibits a distinct translation rate, which can be tightly controlled, leading to significant impacts on protein output per mRNA molecule.
In this webinar, Dr. Jeff Coller will highlight research that shows how leveraging our understanding of mRNA translational regulation aids in the development of innovative disease-modifying treatments for haploinsufficiencies.
A live Q&A session followed the presentation, offering you a chance to pose questions to our expert panelist.
Professor of RNA Biology and Therapeutics
Johns Hopkins University
Webinar produced with support from:
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