Antisense Oligonucleotide Therapy: From Design to the Huntington Disease Clinic
Morgan E. Rook, Amber L. Southwell
Topics & Concepts
Huntington's diseaseHuntingtinDiseaseHuntingtin ProteinSpinal muscular atrophyMedicineClinical trialGenetic enhancementBioinformaticsNeuroscienceBiologyPathologyGeneGeneticsGenetic Neurodegenerative DiseasesMuscle Physiology and DisordersRNA Research and Splicing