Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases
David Barrett, Paula M. Cannon, Federico Mingozzi, Matthew H. Porteus, Isabelle Rivière, Terence R. Flotte
Topics & Concepts
TimelineClinical trialGenetic enhancementTransformative learningCell therapyMedicineBioinformaticsBusinessCellGeneBench to bedsideAction (physics)Intensive care medicineBiotechnologyMEDLINEClinical PracticeGenome editingCRISPR and Genetic EngineeringBiotechnology and Related FieldsBiomedical Ethics and Regulation