Final Program - American Society of Gene & Cell Therapy

Program Schedule, Friday, May 17, 2013
PANEL
Thomas W. Chalberg, Jr., PhD
Artur Isaev, MD, MBA
David Kirn, MD
Sander van Deventer, MD, PhD
Samuel C. Wadsworth, PhD
Scientific Symposium 315
9:45 am - 11:45 am
ROOM: BALLROOM C
Target Non Coding RNAs with Oligonucleotides
CO-CHAIRS: Kevin V. Morris, PhD and Marco S. Weinberg, PhD
SPEAKERS
Claes Wahlestedt, MD, PhD
RNA-Based Strategies To Upregulate Gene Expression
Recently, key roles for regulatory long noncoding RNAs (lncRNAs) in the regulation of gene expression have emerged. I will review
current understanding of the mechanisms of action of lncRNAs and their roles in disease, focusing on recent work in the design
of inhibitors of the natural antisense transcript (NAT) class of lncRNAs, known as AntagoNAT oligonucleotides, and the issues
associated with their therapeutic application.
Christine Esau, PhD
Targeting microRNAs in Human Disease: miR-33 in the Treatment of Atherosclerosis
microRNAs are regulators of the genome that can modulate the activity of speciic gene networks. Single stranded oligonucleotides
can be designed to inhibit the activity of individual microRNAs that are overexpressed in human disease. For example, miR-
33 is a microRNA that is known to be overexpressed in macrophages that may be involved in the formation of atherosclerotic
plaques. Inhibition of miR-33 has been shown de-repress known target mRNAs such as ABCA1, increase cholesterol eflux
from macrophages and regress atherosclerotic lesions in rodent models. Thus targeting microRNAs holds great promise for the
treatment of human diseases.
Friday, May 17, 2013
Keith Gagnon, PhD
Long Noncoding RNAs Link Transcriptional Regulation of Inlammatory Pathway Genes
Coordinated expression of the eicosanoid signaling pathway genes COX-2 and PLA2G4A are regulated by a network of non-coding
RNAs and gene looping. The long non-coding RNAs overlapping the COX-2 gene promoter can be targeted by complementary
endogenous and synthetic small RNAs to elicit activation of both COX-2 and PLA2G4A transcription. This activation requires RNAi
factors like Argonaute 2 and GW182, recruitment of RNA Polymerase II, and chromatin modifying factors. These indings suggest
that non-coding RNA has the potential to assemble related genes into functional operons for regulation and small RNAs can control
this mechanism to synchronously activate transcription.
Kevin V. Morris, PhD
Long non-coding RNAs, Epigenetics and Gene Expression
In human cells some non-coding RNAs have been found to transcriptionally modulate gene expression by targeting epigenetic
complexes to particular loci. The targeted disruption of these suppressor non-coding RNAs can result in targeted activation of
gene expression. Examples of this form of gene activation in disease models ranging from cystic ibrosis to HIV to cancer will be
discussed.
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Final Program SALT LAKE CITY, UTAH May 15–18, 2013