- Cristina R. Reschke, Luiz F.A. Silva, Vamshidhar R. Vangoor, Massimo Rosso, Bastian David, Brenton L. Cavanagh, Niamh M.C. Connolly, Gary P. Brennan, Amaya Sanz-Rodriguez, Catherine Mooney, Aasia Batool, Chris Greene, Marian Brennan, Ronan M. Conroy, Theodor Rüber,Jochen H.M. Prehn, Matthew Campbell, R. Jeroen Pasterkamp, and David C. Henshall
- Targeting coding and noncoding RNAs offers unprecedented potential for precision therapeutics and disease modification. Among leading strategies is the use of antisense oligonucleotides (ASOs), which are now clinically approved for certain neuromuscular diseases and in trials for other conditions. There has been less progress, however, with deployment of ASOs for brain diseases. This is in part due to the blood-brain barrier (BBB) preventing the passage of macromolecules
from the systemic circulation into the brain. Overcoming the BBB may be possible using encapsulation techniques or conjugation to cell-penetrant peptides; however, the safety and efficacy of these approaches remain uncertain.
- MicroRNAs (miRNAs) have recently emerged as a potential therapeutic target in epilepsy. They are small noncoding RNAs that post-transcriptionally reduce protein levels via base-pairing to complementary regions in target mRNAs. In the brain, miRNAs are required for cell growth, differentiation, and synaptic plasticity as well as the control of neuroinflammation and apoptosis.
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