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GENE THERAPY FOR SICKLE CELL DISEASE

GENE THERAPY FOR SICKLE CELL DISEASE
镰状细胞病的基因治疗
批准号：
8150164
负责人：
Punam Malik
金额：
$20.93万
依托单位：
CINCINNATI CHILDRENS HOSP MED CTR
依托单位国家：
美国
项目类别：
财政年份：
2010
资助国家：
美国
项目状态：
已结题
起止时间：
2010-04-01 至 2012-03-31
来源：
https://reporter.nih.gov/project-details/8150164
关键词：
AccountingAffectAllogenicAnemiaAutologousAzacitidineBone MarrowButyratesCD34 geneCellsChromatinClinicClinicalClinical ResearchCodon NucleotidesCollectionDecitabineElementsEngraftmentEquilibriumErythroidErythroid CellsErythropoiesisFanconi&aposs AnemiaFetal HemoglobinGene TransferGenesGeneticGlobinGoalsHarvestHematopoietic stem cellsHemoglobin F DiseaseHumanIn VitroLaboratoriesLentivirus VectorLifeMalignant NeoplasmsMediatingModelingMusPatientsPeripheral Blood Stem CellPharmaceutical PreparationsPhasePhenotypeProductionProtocols documentationRNA InterferenceRecombinantsReportingSafetySickle Cell AnemiaSickle HemoglobinSourceStem cell transplantStem cellsThalassemiaTherapeuticTimeToxic effectbeta Globinbeta Thalassemiachemotherapyconditioningdesigngene therapygene therapy clinical studygenetic manipulationhybrid genehydroxyureaimprovedin vivo Modelpilot trialpolymerizationpre-clinicalpreventsafety studysicklingsmall hairpin RNAvector

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