FINAL PROGRAM - American Society of Gene & Cell Therapy

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13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 33Program ScheduleEducation Session 1203:30 pm - 5:00 pmRoom: Virginia SuiteEmerging Field Review: Genomic Basis of Human DiseaseThis program will be presented by three established investigators at the NIH National Human Genome Research Institute and will highlight active research programs ofdirect relevance to the field of gene and cell based therapies. The topics to be covered include: 1) The Genomics Landscape Circa 2010, 2) The ClinSeq Project: PilotingGenome Sequencing for Research in Genomic, and 3) The NIH Undiagnosed Diseases Program.Wednesday, May 19 thChairPaul B. McCray, Jr., MDSpeakersEric D. Green, MD, PhDThe Genomics Landscape Circa 2010The Human Genome Project’s completion of the human genome sequence in 2003 was a scientific achievement of historic proportions. It also signified a critical transition,as this new foundation of genetic information started to be used in powerful ways by researchers and clinicians to tackle increasingly complex problems in biomedicine.Current efforts in genomics research are focused on using genomic data and technologies to acquire a deeper understanding of biology and to uncover the genetic basis ofhuman disease. Together, these pursuits are moving us down an exciting path towards genomic medicine and fulfilling the promise of a sequenced human genome.Leslie G. Biesecker, MDLarge Scale Sequencing and Hypothesis-Generating Clinical ResearchTo date, high-throughput DNA sequencing has been used to elucidate the complete sequence of various species’ genomes. In contrast, its application to clinically directedresearch has been minimal. It is postulated that this technology will be applied to clinical care in the future – such as for pre-symptomatic diagnoses of medical conditions,customization of preventive strategies, and determination of individuals’ pharmacogenetic profiles. The National Human Genome Research Institute (NHGRI) has a longtermgoal of developing the technology and infrastructure for performing large-scale medical sequencing (LSMS) for health research and, eventually, routine clinical care.Thus, at this time pilot studies are needed to start addressing some of the issues surrounding the development and implementation of LSMS in the clinical research setting.We have launched a pilot project, called ClinSeq, within the NHGRI intramural program. The objective of ClinSeq is to pilot approaches to the technical, medical,and genetic counseling issues related to LSMS by generating a large amount of sequence data from a set of participants. Unlike other projects that maintain anonymizedsamples, ClinSeq is being designed to maintain identified samples to allow us to implement an iterative clinical research strategy that over time will lead to return of individualresults to study participants. With this design we hope not only to contribute to the knowledge about susceptibility genes, but also to understand how to integratethis technology into clinical practice.William A. Gahl, MD, PhDThe NIH Undiagnosed Diseases ProgramThe NIH Undiagnosed Diseases Program (UDP) has as its goals to provide answers to patients with mysterious conditions that have long eluded diagnosis and to advancemedical knowledge about rare and common diseases. In the past two years, the UDP has received 2700 inquiries and 1100 medical records. Approximately 240 patientshave been accepted; a diagnosis has been obtained for 10-15% of patients seen. The application of state-of-the-art genetic analysis makes this program a model for theuse of genetics in the investigation of diagnostic dilemmas. One new disease has been discovered, along with unusual variants of known diseases. The Program alsoprovides insights into the current practice of medicine in the United States.
34American Society of Gene & Cell TherapyProgram ScheduleWednesday, May 19 thEducation Session 1213:30 pm - 5:00 pmRoom: Marriott Ballroom Salon 1Emerging Field Review: Regulatory RNAsAbout a decade ago, it became clear that RNA has a widespread, general role in gene regulation. At least part of this regulation is mediated through a mechanism termedRNA interference (RNAi). The first talk will discuss the basic principles of RNAi and the biology of the small RNA species which mediate RNAi. The discovery of RNAi hasopened up new avenues in genetic engineering which will be covered by the subsequent talks: Delivery of a small interfering RNA (siRNA) or an artificial microRNA(miRNA) with a specific sequence directed against an endogenous mRNA can result in potent knockdown of any gene of interest. Conversely, endogenous regulatoryRNAs can be exploited as an additional layer of regulation to restrict the expression of an exogenously introduced transgene to cells of a specific tissue origin, lineage ordifferentiation state. Finally, as miRNAs are increasingly being implicated in human disease, technologies that allow their manipulation (knockdown and overexpression)become relevant.Co-ChairsBernhard Rudolf Gentner, MD & Alfred Lewin, PhDSpeakersBrian D. Brown, PhDThe Biology and Application of Regulatory RNAsOver the past few years major advances have been made in understanding the action and mechanism of regulatory RNAs. These advances have helped to identify someof the important factors that influence microRNA and target interaction, which, in turn, is facilitating the improvement of technologies that utilize or target the microRNApathway, including RNA interference (RNAi). Here we will discuss some of the recent findings in regulatory RNA biology and how these findings are being applied.Ryan L. Boudreau, PhDRNAi-based Strategies for Silencing DiseaseWith a better understanding of endogenous miRNA biogenesis and gene silencing processes, scientists have devised strategies to co-opt the cellular RNAi machinery inorder to specifically silence various genes of interest. In this way, RNAi serves as a powerful molecular tool used to study gene function in biological processes and providesa novel strategy to treat a variety of diseases (e.g. dominant genetic disorders, cancer and viral invasion, among others). The application of RNAi as a biological ortherapeutic tool is primarily limited by our ability to introduce inhibitory RNAs into target cells or tissues. Inhibitory RNAs can be designed to mimic primary miRNA stemloops(artificial miRNAs), processed pre-miRNAs (short-hairpin RNAs or shRNAs) or mature miRNAs with perfect complementarity to their targets (small interfering RNAs orsiRNAs). This presentation will overview these RNAi vectors, delivery strategies, and highlight important results regarding RNAi vector safety.Bernhard Rudolf Gentner, MDHow to Study and Exploit microRNAs for Gene TherapyChromatin state and the presence of a particular set of transcription factors binding to the promoter sequence determines the transcription of a gene into mRNA. Morerecently, it has become clear that the mRNAoma is extensively subjected to negative regulation by a system involving hundreds of small RNA molecules, the so called“microRNAs”. Similar to the transcription factor complement, the “microRNAoma” is specific for tissues, lineages as well as cellular differentiation and activation states,offering an opportunity to exploit this endogenous layer of regulation to tune the expression of engineered DNA. microRNA regulation has proved a powerful tool toshut down expression in entire tissues like hematopoietic cells, hepatocytes, muscle, and nervous tissue, and thus control transgene immune responses, limit off-targettransgene toxicity and even change the tropism of oncolytic viruses. Another emerging application for microRNA regulated expression cassettes is stem cell research andtherapy. microRNAs allow to dissect different stages of differentiation, from the bona fide stem cell down to the progeny of different lineages. Applications for iPS cells andhematopoietic stem cells will be discussed. A potential side effect of exploiting miRNA regulation is the saturation of miRNA activity and thus deregulation of natural miRNAtargets. Guidelines how to avoid miRNA saturation will be presented.EXHIBITOR PROSPECTUSfinal program
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