RNA therapeutics for epilepsy: An emerging modality for drug discovery

Area of Science:

• Neuroscience
• Genetics
• Pharmacology

Background:

• Epilepsy treatment has historically relied on antiseizure medications (ASMs) discovered through phenotypic screening.
• Current ASMs are effective but do not eliminate drug resistance or offer disease modification.
• Novel therapeutic strategies are crucial for addressing unmet needs in epilepsy management.

Purpose of the Study:

• To explore RNA-based therapeutics as a novel modality for epilepsy treatment.
• To discuss strategies for modulating disease-associated RNA molecules.
• To highlight the potential of RNA therapeutics in various epilepsy patient populations.

Main Methods:

• Review of current literature on RNA therapeutics in epilepsy.
• Categorization of RNA therapeutics targeting noncoding RNAs (ncRNAs) and messenger RNAs (mRNAs).
• Analysis of therapeutic potential for sporadic, drug-resistant, and monogenic childhood epilepsies.

Main Results:

• RNA therapeutics demonstrate potential for disease modification and overcoming drug resistance in epilepsy.
• Targeting ncRNAs shows promise for sporadic, nongenetic epilepsies by modulating disease pathways.
• Targeting mRNAs appears more suitable for monogenic childhood epilepsies.

Conclusions:

• RNA-based drugs represent a promising new frontier in epilepsy treatment.
• Modulating specific RNA molecules offers tailored therapeutic strategies for different epilepsy types.
• RNA therapeutics hold potential for treating drug-resistant and genetic forms of epilepsy.