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preclinical Testing of Novel Therapies in FA

preclinical Testing of Novel Therapies in FA
FA 中新疗法的临床前测试
批准号：
8255537
负责人：
Markus Grompe
金额：
$30.17万
依托单位：
OREGON HEALTH & SCIENCE UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2011
资助国家：
美国
项目状态：
已结题
起止时间：
2011-04-01 至 2015-03-31
来源：
https://reporter.nih.gov/project-details/8255537
关键词：
Acute Myelocytic LeukemiaAndrogen ReceptorAndrogensAnemiaBiological AssayBone MarrowCafe-au-Lait SpotCause of DeathCellsClinicalCongenital AbnormalityDiseaseDrug usageFailureFanconi&aposs AnemiaFunctional disorderGene ExpressionGene Transduction AgentGenesHematopoiesisHematopoieticHematopoietic stem cellsHemorrhageHumanIn VitroKnockout MiceLongevityMalignant NeoplasmsMarrowMeasuresMediatingMethodsMicrophthalmosModelingMolecularMusOrganOxidantsOxymetholonePancytopeniaPatientsPharmaceutical PreparationsPluripotent Stem CellsPreclinical TestingRadialRare DiseasesResveratrolScreening procedureSignal PathwaySolidSolid NeoplasmSquamous cell carcinomaStem cellsSuperoxide DismutaseTestingTherapeuticThioctic AcidThumb structureadeno-associated viral vectoranalogcancer preventionclinically relevantefficacy testinggene discoverygene repairgene therapyimprovedin vivomutantnovelpreventprogenitorsmall moleculestemtempoltumor

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中文摘要
翻译
FA是一种罕见的疾病，在美国约有500名FA患者存活。FA的3个主要临床特征是出生缺陷（拇指/桡骨缺失;小眼症;咖啡Au-lait斑等），进行性骨髓衰竭和癌症易感性[1]。在FA中。寿命大大缩短[2]。死亡的主要原因是骨髓衰竭，并伴有贫血、出血和感染等并发症。另一种死亡原因是癌症，特别是急性髓性白血病，但也有实体瘤，特别是鳞状细胞癌[3]。
英文摘要
FA is a rare disease and about 500 patients with FA are alive in the U.S.A. The 3 main clinical features of FA are birth defects (missing thumb/radius; microphthalmia; cafe-au-lait spots etc.), progressive marrow failure and cancer suscepfibility [1]. In FA. the life span Is severely shortened [2]. The main cause of death is bone marrow failure with the associated complications of anemia, bleeding and infecfion. Another cause of death is cancer, particulariy acute myelogenous leukemia, but also solid tumors, particulariy squamous cell carcinomas [3].
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