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bluebird bio
美國bluebirdbiowww.bluebirdbio.com單基因突變基因療法bluebirdbio isdevelopinginnovativegenetherapiesforseveregeneticdisorders. Attheheartofbluebirdbio’sproductcreationeffortsisitsbroadlyapplicablegenetherap yplatformforthedevelopmentofnoveltreatmentsfordiseaseswithfewornoclinicaloptions. Thecompany’snovelapproachusesstemcellsharvestedfromthepatient’sbonemarrowintowhichahealthyversionofthediseasecausinggeneisinserted. Afterbeinggrowninculture,thosecellsaregivenbacktothepatient. bluebirdbio’sapproach representsatrueparadigmshiftinthetreatmentofseveregeneticdiseasesbyeliminatingthepotentialcomplicationsassociatedwithdonorcelltransplantationandpotentiallypresentingaone-timetransformativetherapy.bluebirdbiohastwolaterstageclinicalproductsindevelopmentforchildhoodcerebraladrenoleukodystrophy(CCALD)
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