RNA therapeutics for neurological diseases

Introduction: Ribonucleic acid (RNA) therapeutics are a new class of drugs whose importance is highlighted by the growing number of molecules in the clinic.

Sources of data: We focus on RNA therapeutics for neurogenetic disorders, which are broadly defined as diseases with a genetic background and with at least one clinical sign affecting the nervous system. A systematic search identified 14 RNA drugs approved by FDA and many others in development.

Areas of agreement: The field of RNA therapeutics is changing the therapeutic scenario across many disorders.

Areas of controversy: Despite its recent successes, RNA therapeutics encountered several hurdles and some clinical failures. Delivery to the brain represents the biggest challenge.

Growing points: The many advantages of RNA drugs make the development of these technologies a worthwhile investment.

Areas timely for developing research: Clinical failures stress the importance of implementing clinical trial design and optimizing RNA molecules to hold the promise of revolutionizing the treatment of human diseases.