G2C4 targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons
Jeffrey D. Rothstein, Victoria Baskerville, Sampath Rapuri, Emma Mehlhop, Paymaan Jafar‐Nejad, Frank Rigo, Frank Bennett, Sarah Mizielinska, Adrian M. Isaacs, Alyssa N. Coyne
Topics & Concepts
C9orf72Trinucleotide repeat expansionInduced pluripotent stem cellSense (electronics)BiologyRNAAmyotrophic lateral sclerosisFrontotemporal dementiaAntisense RNAGeneticsGeneMedicineDementiaDiseaseChemistryPathologyPhysical chemistryEmbryonic stem cellAlleleAmyotrophic Lateral Sclerosis ResearchNeurogenetic and Muscular Disorders ResearchGenetic Neurodegenerative Diseases