Final Program - American Society of Gene & Cell Therapy

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Program Schedule, Wednesday, May 15, 2013 Scientific Symposium 105 8:00 am - 10:00 am ROOM: 150 ABC Polymeric Gene Delivery CHAIR: Sung Wan Kim, PhD SPEAKERS Wim E. Hennink, PhD Decationized Polyplexes: Novel Polymeric Gene Delivery System with Reduced Toxicity The clinical applicability of polymers as gene delivery systems depends not only on their eficiency, but also on their safety. The cytotoxicity of these systems remains a major issue, mainly due to their cationic nature. Therefore, it is highly preferable to have a system based on biocompatible neutral polymers, lacking polycations, without compromising the DNA condensing and protecting capacities. We introduce a concept to obtain a neutral polymeric gene delivery system, through a 3-step process to generate polyplexes with a core of disulide crosslinked poly(hydroxypropyl methacrylamide) (pHPMA) in which plasmid DNA (pDNA) is entrapped and a shell of poly(ethylene glycol) (PEG). Wednesday, May 15, 2013 Ram I. Mahato, PhD Emerging Trends in Combination Therapy with RNAi Treatment strategies relying on a single small molecule drug, or silencing of a single gene often fail, while simultaneous targeting of multiple cellular pathways is more likely to succeed. RNA interference (RNAi) is a promising approach for treating diabetes and cancer. By pooling siRNAs targeting two different regions of the same target gene, or combining two siRNAs against two different genes, or even co-formulating small molecule drug with speciic siRNA or miRNA, we hope to improve islet transplantation and treat advanced prostate and pancreatic cancer. In this presentation, I will discuss our recent results on RNAi technologies for treating diabetes and cancer. Chae-Ok Yun, PhD Harnessing Adenovirus for Tumor Targeting by Nanocomplex A challenge to develop adenovirus (Ad)-mediated therapeutics has been issued to treat metastatic cancer via systemic administration. For effective gene therapeutics against primary and metastatic lesions, a systemically injectable tumor-targeting Ad vector system must be developed. Systemic delivery of Ad, however, is hampered by immune response against Ad, short half-life in the circulation, liver uptake, and low accumulation at target disease sites. Modiication of the Ad surface allows Ad to circulate in the bloodstream for a longer time, to be not targeted to the liver, and to passively accumulate in tumor sites via enhanced permeation and retention effects. The addition of afinity tags results in active targeting and high eficacy. Strategies including addition of polymer complexes, chemical modiications, and targeting moieties for the development of systemically injectable Ad gene therapeutic carriers will be discussed. Kazunori Kataoka, PhD Polymeric Micellar Nanocarriers for Gene and Oligonucleotide Delivery Focus of my talk will be on the use of self-assembly structures from multi-functional block copolymers, particularly polymeric micelles, for gene and oligonucleotide delivery. Importance of peptide ligands to facilitate extravasation as well as to control intracellular traficking of polymeric micelles will be discussed. Also, I will present our new strategy to delivery messenger RNA to target organ. Final Program SALT LAKE CITY, UTAH May 15–18, 2013 35
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. 36 Final Program SALT LAKE CITY, UTAH May 15–18, 2013
Page 2 and 3: ASGCT Officers and Board of Directo
Page 4 and 5: Annual Meeting Exhibitors - as of A
Page 6 and 7: Annual Meeting Supporters Patron Le
Page 8 and 9: Committee Rosters Advisory Council
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Page 12 and 13: Committee Rosters Program Committee
Page 14 and 15: Abstract Reviewers Cancer - Immunot
Page 16 and 17: Outstanding New Investigator Awards
Page 18 and 19: Faculty List Thomas W. Chalberg, Jr
Page 20 and 21: Faculty List Ann M. Leen, PhD Baylo
Page 22 and 23: Faculty List Evan Y. Snyder, MD PhD
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Abstract Author Index Aartsma-Rus,
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Final Program - American Society of Gene & Cell Therapy

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