FINAL PROGRAM - American Society of Gene & Cell Therapy

More documents

Recommendations

Info

13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 35Program ScheduleEducation Session 1223:30 pm - 5:00 pmRoom: Wilson ABCTopical Review: Cell TherapyChairEdwin M. Horwitz, MD, PhDSpeakersEdwin M. Horwitz, MD, PhDFundamentals of Cell TherapyPeter Johnston, MD, FACCStem Cell Therapy for the HeartNearly 6 million people in the United States suffer from heart failure. This burden of disease has driven interest in stem cell therapy for the heart in the hope that dysfunctionalmyocytes and vasculature might be repaired or replaced. Clinical trials to date have explored the use of various skeletal, bone marrow-derived, endothelial, andendogenous cardiac precursor cells for cardiac repair. Along with much promise, there remain many questions about not only the optimal type of cell, but also the timingand delivery method necessary to achieve the maximal therapeutic benefit.Dao Pan, PhDCell and Gene Therapy for CNS Abnormalities in Lysosomal Storage DisordersLysosomal storage diseases (LSD) are a group of ~40 inherited metabolic disorders with a combined incidence of 1 in 1500 to 7000 live births. More than 70% of allLSD affects the central nervous system (CNS). Bone marrow stem cell transplantation has been shown to ameliorate many visceral symptoms in some LSD patients;however CNS disease associated with LSD remains as a major challenge. Here we will discuss experimental and preclinical studies toward new therapeutic strategies,including stem cell-based brain transplantation therapies, hematopoietic stem cell gene therapies, and CNS-targeted gene therapies. Issues that are pertinent to theirpotential translational applications will also be addressed.Wednesday, May 19 thEducation Session 1233:30 pm - 5:00 pmRoom: Thurgood Marshall WTopical Review: Gene Therapy for Eye DiseasesRecent successes in viral vector-based gene therapy clinical trials underscore the potential of gene therapy as a means to provide definitive solutions for currently incurableocular diseases. This session will focus on the basic biology and vector development for ocular diseases. In addition, it will provide recent updates on novel strategies invector design, as well as the status and prospects of gene therapy clinical trials for ocular diseases.Co-ChairsPeter A. Campochiaro, MD & Tal Kafri, PhDSpeakersBill Hauswirth, PhDAAV Vector Considerations for the RetinaConsiderations for AAV vectors intended for transducing cells of the retina include 1) the anatomical site of vector injection, 2) the vector serotype, 3) the size of thevector genome, 4) the strandedness of the vector genome and 5) the ability of novel AAV vectors to penetrate retinal tissue. Each issue will be discussed in the context ofgene-based therapies for degenerative retinal diseases.John G. Flannery, PhDEvolution of AAV Vectors for Ocular Gene Therapy
36American Society of Gene & Cell TherapyProgram ScheduleWednesday, May 19 thPeter A. Campochiaro, MDOverview of Gene Therapy for Ocular DiseasesThe potential of ocular gene therapy has been demonstrated in animal models and early clinical trials. Proof-of-concept has been provided for gene replacement of mutatedgenes in inherited retinal degenerations by 3 clinical trials demonstrating improved visual function by replacement of the RPE65 gene in patients with Leber’s congenitalamaurosis. Studies are being considered for X-linked retinoschisis and Stargardt’s disease. A trial in which an antiangiogenic protein was delivered with an adenoviralvector for neovascular age-related macular degeneration (AMD) has shown good safety and some suggestion of biological activity. A trial in which a vascular endothelialgrowth factor binding protein is delivered with an AAV vector in patients with neovascular AMD has recently started. Preclinical studies suggest that gene delivery of neurotrophicfactors or antioxidant proteins may be considered for retinal degenerations. Thus early clinical trials suggest several promising avenues for ocular gene therapy.Education Session 1243:30 pm - 5:00 pmRoom: Maryland SuiteTopical Review: Stem Cell BasicsIn order for stem cells to fulfill their potential as viable cell transplant/gene transfer therapeutics, it will be necessary to understand the process of stem cell self-renewalto obtain sufficient cell numbers to be applicable for transplant. Additionally, the genes/factors involved in differentiating these pluripotent cells down specific lineagepathways will also be crucial in the development of a transplantable therapeutic. The speakers in this session will address these important issues that not only providefurther insight into organ and tissue development but also open up therapeutic avenues of practical application of these cells in regenerative medicine.ChairWilliam F. Goins, PhDSpeakersG. Ian Gallicano, PhDStem Cells: Past, Present, FutureThe idea of stem cell based therapies is particularly prevalent in the fields of diabetes, cardiac and neurological disorders. However, while the field of stem cell therapiesmay be relatively new, the road for stem cell research has been long and winding. Understanding hoe the evolution of the stem cell field has led to its current state isnot only interesting, it can serve as a powerful tool for predicting its future. Consequently, this presentation will focus on the basic evolution of stem cells and their use intherapies.David F. Stroncek, MDGene and MicroRNA Expression in Human Embryonic Stem CellsGlobal gene expression analysis has been used to identify factors responsible for the pluripotency of human embryonic stem (hES) cells. Most genes expressed by hES areinvolved with RNA post-transcriptional modification, DNA replication, recombination and repair, RNA Damage repair, cell cycle, and cell growth and proliferation. Transcriptfactors are among the most highly expressed hES genes. Studies focusing on specific genes found that the transcription factors OCT4, NANOG and SOX2 are important inhES development and pluripotency. Transcription factors likely interact with chromatin remodeling factors and histone modifying enzymes to modulate chromatin conformation.The knockout of dicer in hES, a nuclease critical in microRNA generation, compromises hES cell proliferation and differentiation and demonstrates an important rolefor microRNA in hES cells. MicroRNA in the miR-302 and miR-520 clusters are expressed in hES cells and may contribute to chromatin structure modifications.Ming Zhan, PhDComputational Biology Studies to Decipher Molecular Mechanisms Controlling Embryonic Stem Cell PluripotencyComputational biology has played a significant role in investigation of embryonic stem cells (ESCs). In this presentation, I will describe comparative genomics andcomparative transcriptomics studies conducted on ESCs and genetic regulatory network analysis of ESCs through mathematical modeling. All the studies contribute to asystems understanding of molecular mechanisms controlling ESC self-renewal and differentiation.Break5:00 pm – 5:30 pmEXHIBITOR PROSPECTUSfinal program
Page 1 and 2: FINAL PROGRAMAmerican Society of Ge
Page 4: 13 th AnnUAL MEETing | Washington,
Page 7 and 8: 6American Society of Gene & Cell Th
Page 12 and 13: 13 th AnnUAL MEETing | Washington,
Page 86 and 87:
13 th AnnUAL MEETing | Washington,
Page 88 and 89:
Page 90 and 91:
Page 92 and 93:
Page 94 and 95:
Page 96 and 97:
Page 98 and 99:
Page 100 and 101:
Page 102 and 103:
Page 104 and 105:
Page 106 and 107:
Page 108 and 109:
Page 110 and 111:
Page 112 and 113:
Page 114 and 115:
Page 116 and 117:
Page 118 and 119:
Page 120 and 121:
Page 122 and 123:
Page 124 and 125:
Page 127 and 128:
126American Society of Gene & Cell
Page 129 and 130:
Page 131 and 132:
Page 133 and 134:
Page 135 and 136:
Page 137 and 138:
Page 139 and 140:
Page 141 and 142:
Page 143 and 144:
Page 145 and 146:
Page 147 and 148:
Page 149 and 150:
Page 151 and 152:
Page 153 and 154:
Page 155 and 156:
Page 157 and 158:
Page 159 and 160:
Page 161 and 162:
Page 163 and 164:
Page 165 and 166:
Page 167 and 168:
Page 169 and 170:
Page 171 and 172:
Page 173 and 174:
Page 175 and 176:
Page 177 and 178:
Page 179 and 180:
Page 181 and 182:
Page 183 and 184:
Page 185 and 186:
Page 187 and 188:
show all

FINAL PROGRAM - American Society of Gene & Cell Therapy

Create successful ePaper yourself

Delete template?

Save as template?