Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation
Xin Chen, Sarah Snanoudj-Verber, Laura Pollard, Yuhui Hu, Sara Cathey, Ritva Tikkanen, Steven J. Gray
Topics & Concepts
Genetic enhancementTranslation (biology)BiologyMedicineGenePharmacologyComputational biologyGeneticsMessenger RNAVirus-based gene therapy researchComplement system in diseasesNeurogenetic and Muscular Disorders Research