Failure of <i>C9orf72</i> sense repeat-targeting antisense oligonucleotides: lessons learned and the path forward
Alexander J. Cammack, Rubika Balendra, Adrian M. Isaacs
Abstract
The recent failure of two independent clinical trials targeting C9orf72 sense repeat-containing RNAs with antisense oligonucleotides was a great disappointment for the field. Cammack et al. discuss the data from these trials, possible reasons for the failures, and the future of C9orf72 therapeutic targeting moving forward.
Topics & Concepts
Sense (electronics)C9orf72OligonucleotidePath (computing)DisappointmentComputer scienceMedicineBiologyPsychologyDiseaseInternal medicineGeneticsEngineeringComputer networkDNADementiaSocial psychologyFrontotemporal dementiaElectrical engineeringNeurogenetic and Muscular Disorders ResearchRNA Research and SplicingRNA regulation and disease