• Ilaria Brentari, Mariia Zadorozhna, Michela Alessandra Denti, and Elisa Giorgio.
• Dipartimento di Biologia Cellulare, Computazionale e Integrata (CIBIO), Università degli Studi di Trento, 38123 Trento, Italy.
• Br. Med. Bull. 2023 Sep 12; 147 (1): 506150-61.

IntroductionRibonucleic acid (RNA) therapeutics are a new class of drugs whose importance is highlighted by the growing number of molecules in the clinic.Sources Of DataWe 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 AgreementThe field of RNA therapeutics is changing the therapeutic scenario across many disorders.Areas Of ControversyDespite its recent successes, RNA therapeutics encountered several hurdles and some clinical failures. Delivery to the brain represents the biggest challenge.Growing PointsThe many advantages of RNA drugs make the development of these technologies a worthwhile investment.Areas Timely For Developing ResearchClinical failures stress the importance of implementing clinical trial design and optimizing RNA molecules to hold the promise of revolutionizing the treatment of human diseases.© The Author(s) 2023. Published by Oxford University Press. All rights reserved. For Permissions, please email: journals.permissions@oup.com.

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