[HTML][HTML] Antisense oligonucleotides: treating neurodegeneration at the level of RNA

SL DeVos, TM Miller - Neurotherapeutics, 2013 - Elsevier
Adequate therapies are lacking for Alzheimer's disease, Parkinson's disease, Huntington's
disease, amyotrophic lateral sclerosis, and other neurodegenerative diseases. The ability to
use antisense oligonucleotides (ASOs) to target disease-associated genes by means of
RNA may offer a potent approach for the treatment of these, and other, neurodegenerative
disorders. In modifying the basic backbone chemistry, chemical groups, and target
sequence, ASOs can act through numerous mechanisms to decrease or increase total …