Antisense oligonucleotide therapy in a humanized mouse model of <i>MECP2</i> duplication syndrome
Yingyao Shao, Yehezkel Sztainberg, Qi Wang, Sameer S. Bajikar, Alexander J. Trostle, Ying‐Wooi Wan, Paymaan Jafar‐Nejad, Frank Rigo, Zhandong Liu, Jianrong Tang, Huda Y. Zoghbi
Abstract
-ASO is therefore well tolerated and beneficial in this mouse model and provides a translatable approach that could be feasible for treating MDS.
Topics & Concepts
Gene duplicationHumanized mouseOligonucleotideMECP2Genetic enhancementMedicineBiologyCancer researchIn vivoGeneGeneticsPhenotypeGenetics and Neurodevelopmental DisordersEnergy Harvesting in Wireless NetworksChromatin Remodeling and Cancer