Current limitations of gene therapy for rare pediatric diseases: Lessons learned from clinical experience with AAV vectors
Antoine Gardin, Giuseppe Ronzitti
Topics & Concepts
Genetic enhancementClinical trialMedicineGene transferAdeno-associated virusVector (molecular biology)PopulationIntensive care medicineBioinformaticsComputational biologyGeneBiologyPathologyGeneticsEnvironmental healthRecombinant DNAVirus-based gene therapy researchCRISPR and Genetic EngineeringViral Infections and Immunology Research