23rd Annual Fanconi Anemia Research Fund Scientific Symposium

第23届年度范可尼贫血研究基金科学研讨会

基本信息

批准号：
8205078
负责人：
Grover Carlton Bagby
金额：
$ 1万
依托单位：
FANCONI ANEMIA RESEARCH FUND, INC.
依托单位国家：
美国
项目类别：
财政年份：
2011
资助国家：
美国
起止时间：
2011-07-15 至 2012-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8205078
关键词：
Acute Affect Aging Animal Model Apoptosis Basic Science Biological Models Blood Cells Candidate Disease Gene Carcinogenesis Mechanism Cell Survival Cell physiology Cells Clinical Research Collaborations Complement Complex Cues DNA Repair Data Defect Development Disease Dyskeratosis Congenita Dysmyelopoietic Syndromes Endocrine Glands Epigenetic Process Epithelial Erythroid Progenitor Cells Face Family Fanconi anemia protein Fanconi&apos s Anemia FarGo Fostering Functional disorder Funding Gene Expression Gene Expression Regulation Genes Genetic Head and Neck Cancer Hematopoiesis Hematopoietic Hematopoietic Neoplasms Hematopoietic Stem Cell Transplantation Hereditary Disease Hypersensitivity Immune response In Vitro Incidence Inflammatory Interdisciplinary Study Laboratory Diagnosis Life Malignant Neoplasms Malignant Squamous Cell Neoplasm Malignant neoplasm of lung Malignant neoplasm of ovary Modeling Molecular Myelogenous Myeloid Leukemia Oral Pancytopenia Pathogenesis Pathway interactions Patients Physicians Plasmacytic Leukemia Pluripotent Stem Cells Population Production Protein Binding Proteins RNA Interference Radiation Rare Diseases Regulation Reporting Research Research Personnel Research Project Grants Schedule Science Screening procedure Signal Pathway Signaling Molecule Solid Neoplasm Spain Stem cell transplant Stem cells Stress Students Survivors Therapeutic Translational Research Tumor Suppression abstracting carcinogenesis clinical Diagnosis crosslink cytokine extracellular gene therapy graduate student high risk homologous recombination in vivo insight leukemogenesis meetings neoplastic cell protein function response senescence small molecule stem cell differentiation symposium tool translational study

项目摘要

DESCRIPTION (provided by applicant): 23rd Annual Fanconi Anemia Research Fund Scientific Symposium Fanconi anemia (FA) is a rare hereditary disease characterized by bone marrow failure, developmental anomalies, a high incidence of myelodysplasia (MDS), acute non-lymphocytic leukemia (AML), solid tumors, and cellular hypersensitivity to cross-linking agents. A unique feature of Fanconi anemia is the development, in early life, of specific epithelial and hematopoietic malignancies usually found only in aging populations. The function of the proteins is largely unknown but many form complexes with each other and in one canonical "pathway," eight of the eleven known Fanconi anemia proteins bind together in a complex and monoubiquitinate FANCD2, one of the proteins not in the "core" complex. There is in vitro and in vivo evidence that at least some of the FA proteins also promote survival signaling pathways in hematopoietic cells by forming complexes with signaling molecules. Stem cell transplantation is the treatment of choice for eligible patients with bone marrow failure. Survivors of bone marrow failure ultimately face an extremely high risk of developing squamous cell cancers. The disease is an ideal candidate for gene therapy because of the inherent selectability of complemented stem cells. Broad evidence is being developed that dysfunction of the FA signaling pathways can develop as somatic changes (epigenetic and genetic) in neoplastic cells arising in non-Fanconi patients and that the consequences of such somatic changes suppress self-replicative potential and enhance senescence in progenitor cell pools. The annual Fanconi Anemia Research Fund Scientific Symposium is a three-day conference comprised of select invited presentations and approximately 35-45 oral abstract presentations (to be chosen from a call for abstracts) in a single-track format. The Symposium brings together leading researchers and physicians as well as young investigators, graduate and post-graduate students from around the world to discuss all basic, translational, and clinical research aspects of this rare disease. The Fanconi Anemia Research Fund actively encourages and supports attendance and submissions by students and junior investigators. The meeting provides a unique opportunity for investigators to cross-fertilize and develop interdisciplinary research projects. Not only will the data be shared freely with all investigators, the files will be made accessible through GeneSifter, an online bio-information tool. This application seeks partial support for this meeting, scheduled to be held in Barcelona, Spain in October 2011.

描述（由申请人提供）：第23届年度Fanconi贫血研究基金科学研讨会Fanconi贫血（FA）是一种罕见的遗传性疾病，其特征是骨髓衰竭，发育异常，骨髓增生的高发病率（MDS），急性非淋巴细胞症（AML），固体肿瘤（AML），固体肿瘤（AML），和固体肿瘤（AML），和对交联药的超敏反应。 Fanconi贫血的一个独特特征是早期生活，特定上皮和造血恶性肿瘤的发展通常仅在衰老人群中。蛋白质的功能在很大程度上是未知的，但是许多彼此形成了复合物，并且在一个规范的“途径”中，其中八个已知的fanconi贫血蛋白蛋白中的八个在复合物和单液化的fancd2中结合在一起，这是“核心”，而不是“核心”中的一种。复杂的。有体外和体内证据表明，至少某些FA蛋白还通过用信号分子形成复合物来促进造血细胞中的存活信号通路。干细胞移植是符合条件的骨髓衰竭患者的选择治疗。骨髓衰竭的幸存者最终面临着出现鳞状细胞癌的极高风险。由于互补的干细胞的固有选择性，该疾病是基因治疗的理想候选者。广泛的证据表明，FA信号通路的功能障碍可以随着非癌症患者产生的肿瘤细胞的体细胞变化（表观遗传和遗传）的发展而发展，并且这种体细胞变化的后果抑制了自我复制潜力并增强了祖细胞细胞衰老的影响游泳池。一年一度的Fanconi贫血研究基金科学研讨会是为期三天的会议，由精选的邀请演讲和大约35-45个口头摘要演示文稿（旨在从摘要的呼吁中选择），以单轨格式为单位。该研讨会汇集了领先的研究人员和医生以及来自世界各地的年轻研究人员，研究生和研究生，讨论这种罕见疾病的所有基本，转化和临床研究方面。 Fanconi贫血研究基金会积极鼓励并支持学生和初级调查人员的出勤和提交。会议为调查人员提供了一个独特的机会，可以交叉利用和开发跨学科研究项目。数据不仅可以与所有调查人员自由共享，还可以通过在线生物信息工具Genesifter访问这些文件。该申请为计划于2011年10月在西班牙巴塞罗那举行的这次会议寻求部分支持。