Final Program - American Society of Gene & Cell Therapy

Program Schedule, Wednesday, May 15, 2013
Wednesday, May 15, 2013
Education Sessions
The goal of the ASGCT Education Program is to provide participants with a broad background and introduction to topics relevant to the
ield of gene and cell therapy. Education Sessions will be subdivided into two headings. “Topical Review” sessions will provide a broad
perspective of an area of importance to the gene and cell therapy community such as major vector systems, speciic target organs or
diseases, and funding/regulatory issues. “Emerging Field Review” sessions will provide a broad perspective of a technological or scientiic
discipline that has recently become of great signiicance to ASGCT members.
Education Session 110
10:30 am - 12:00 pm
ROOM: BALLROOM B
Emerging Field Review: Gene Targeting
Gene targeting, also known as genome editing, has become an increasingly feasible gene therapy strategy. In this education, three pioneers
in the ield will describe different approaches to gene targeting. After this session, attendees should be able to understand the different
approaches to gene targeting and which strategy might be best for their speciic gene therapy application.
CHAIR: Matthew H. Porteus, MD, PhD
SPEAKERS
David W. Russell, MD, PhD
AAV-Mediated Gene Targeting
This presentation will describe the mechanism and applications of AAV-mediated gene targeting, which has been used to target
over 100 genes in more than 50 different cell types. Sequence changes can be introduced at high frequencies, with up to 1% of
infected cells undergoing gene targeting. Insertions, deletions and substitutions can all be accurately introduced, and additional
unwanted mutations are not found at targeted loci. Site-speciic nucleases are not required to obtain high targeting frequencies, so
there is no risk of introducing DNA breaks at off-target sites.
J. Keith Joung, MD, PhD
Engineered TALENs and CRISPR-Cas RNA-Guided Nucleases (RGNs) for Targeted Genome Editing
Engineered site-speciic nucleases have emerged as a broadly applicable technology for eficiently inducing targeted alterations
in the genomes of living cells and organisms. This talk will provide an introduction to two engineered nuclease platforms:
transcription activator-like effector nucleases (TALENs) and CRISPR-Cas RNA-Guided Nucleases (RGNs). The strengths and
limitations of each of these platforms will be discussed. In addition, publicly available methods and resources for practicing these
technologies will be reviewed.
Dana Carroll, PhD
Nuclease-mediated Gene Targeting
With new and rapidly developing technology, it is now possible to make knock-outs and knock-ins in virtually any gene in a wide
range of cells and organisms. The secret is the development of nucleases that can be designed to cleave arbitrarily chosen genomic
DNA sequences with high speciicity. Cellular repair processes create localized mutations at the nuclease-induced break, or copy
novel sequences from a user-provided template. This talk will cover two classes of targetable cleavage reagents, zinc-inger
nucleases (ZFNs) and TALENs. The research and therapeutic potential of these reagents is enormous, and ZFNs are currently in
clinical trials.
Education Session 111
10:30 am - 12:00 pm
ROOM: BALLROOM C
Emerging Field Review: Ocular Gene Therapy: Tools and Genes
Following the success of the clinical gene therapy trials for an early onset form of inherited blindness called LCA2, the momentum in the
ield of ocular gene therapy is palpable. In this session, we try to capture this excitement by providing an in depth review into the genetics
of vision and blindness and the technologies that are being developed to therapeutically intervene in the disease course using gene transfer.
This educational session also will discuss disease gene discovery in the genomic era, genetic diagnosis for inherited forms of blindness, gene
therapies for blindness under development, and AAV and nanoparticle vector systems for retinal targeting.
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Final Program SALT LAKE CITY, UTAH May 15–18, 2013