RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model
Osasumwen V. Aimiuwu, Allison M. Fowler, Megha Sah, Jia Jie Teoh, Ayla Kanber, Nettie K. Pyne, Sabrina Petri, Chana Rosenthal-Weiss, Mu Yang, Scott Q. Harper, Wayne N. Frankel
Topics & Concepts
RNA interferenceHypotoniaBiologyGenetic enhancementEpilepsyGeneDiseaseBioinformaticsEncephalopathyMedicineGeneticsNeuroscienceInternal medicineRNAGenetics and Neurodevelopmental DisordersRNA regulation and diseaseRNA modifications and cancer