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Review
. 2023 Sep 12;147(1):50-61.
doi: 10.1093/bmb/ldad010.

RNA therapeutics for neurological diseases

Ilaria Brentari  1 , Mariia Zadorozhna  2 , Michela Alessandra Denti  1 , Elisa Giorgio  2   3
Affiliations
Review

RNA therapeutics for neurological diseases

Ilaria Brentari et al. Br Med Bull. .

Abstract

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.

Keywords: RNA drugs; RNA therapeutics; genetic diseases; innovative therapeutic options; neurological diseases.

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