Targeting the Root Cause of Mucopolysaccharidosis IIIA with a New scAAV9 Gene Replacement Vector
Tierra A. Bobo, Preston N. Samowitz, Michael I. Robinson, Haiyan Fu
Abstract
vector genomes (vg)/kg was functionally effective. Furthermore, the vector treatment did not lead to detectable systemic toxicity or adverse events in MPS IIIA mice. These data demonstrate the development of a safe and effective new gene therapy product for treating MPS IIIA, which further support the extended clinical relevance of platform recombinant AAV9 (rAAV9 gene delivery for treating broad neurogenetic diseases.
Topics & Concepts
Vector (molecular biology)Genetic enhancementAdeno-associated virusEnzyme replacement therapyMedicineViral vectorMucopolysaccharidosisRecombinant DNAAdverse effectGene deliveryBiologyVirologyPharmacologyImmunologyGeneInternal medicineDiseaseGeneticsLysosomal Storage Disorders ResearchNeurogenetic and Muscular Disorders ResearchVirus-based gene therapy research