Ex Vivo and In Vivo Gene Therapy for Mucopolysaccharidoses: State of the Art
Giulia Consiglieri, Maria Ester Bernardo, Nicola Brunetti‐Pierri, Alessandro Aiuti
Topics & Concepts
Ex vivoEnzyme replacement therapyMedicineGenetic enhancementIn vivoClinical trialHematopoietic stem cell transplantationTransplantationDiseaseBioinformaticsImmunologyInternal medicineGeneBiologyBiotechnologyGeneticsLysosomal Storage Disorders ResearchVirus-based gene therapy researchNeurogenetic and Muscular Disorders Research