Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue
Gretchen M. Thomsen, Arthur H.M. Burghes, Caroline Hsieh, Janet Do, Binh T. T. Chu, Stephanie T. Perry, Basam Z. Barkho, Petra Kaufmann, Douglas M. Sproule, Douglas E. Feltner, Wendy K. Chung, Vicki L. McGovern, Robert F. Hevner, Miriam Conces, Christopher R. Pierson, Mariacristina Scoto, Francesco Muntoni, Jerry R. Mendell, Kevin D. Foust
Topics & Concepts
Spinal muscular atrophyGenetic enhancementBiodistributionSMN1BiologyMotor neuronCentral nervous systemGene expressionPathologyMedicineInternal medicineEndocrinologyGeneDiseaseIn vivoGeneticsNeurogenetic and Muscular Disorders ResearchCongenital Anomalies and Fetal SurgeryRNA modifications and cancer