Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice
Hannah G. Lahey, Chelsea Joy Webber, Diane Golebiowski, C Izzo, Erin Horn, Toloo Taghian, Paola Rodrı́guez, Ana Rita Batista, Lauren E. Ellis, Misako Hwang, Douglas R. Martin, Heather Gray‐Edwards, Miguel Sena‐Esteves
Topics & Concepts
Sandhoff diseaseHEXAGangliosidosisLysosomal storage diseaseTay-Sachs diseaseGenetic enhancementGangliosideHexosaminidaseVector (molecular biology)BiologyPharmacologyImmunologyVirologyMedicineInternal medicineDiseaseEnzymeGeneGeneticsBiochemistryRecombinant DNALysosomal Storage Disorders ResearchVirus-based gene therapy researchCytomegalovirus and herpesvirus research