Nucleic acid-based therapeutics for the treatment of central nervous system disorders

Robyn McCartan; Olga Khorkova; Claude-Henry Volmar; Claes Wahlestedt

doi:10.3389/fgene.2023.1250276

Nucleic acid-based therapeutics for the treatment of central nervous system disorders

Front Genet. 2023 Aug 16:14:1250276. doi: 10.3389/fgene.2023.1250276. eCollection 2023.

Authors

Robyn McCartan^{1

2}, Olga Khorkova^{1

3}, Claude-Henry Volmar^{1

2}, Claes Wahlestedt^{1

2}

Affiliations

¹ Center for Therapeutic Innovation, University of Miami Miller School of Medicine, Miami, Florida, United States.
² Department of Psychiatry and Behavioral Sciences, University of Miami Miller School of Medicine, Miami, Florida, United States.
³ OPKO Health, Miami, Florida, United States.

Abstract

Nucleic acid-based therapeutics (NBTs) are an emerging class of drugs with potential for the treatment of a wide range of central nervous system conditions. To date, pertaining to CNS indications, there are two commercially available NBTs and a large number of ongoing clinical trials. However, these NBTs are applied directly to the brain due to very low blood brain barrier permeability. In this review, we outline recent advances in chemical modifications of NBTs and NBT delivery techniques intended to promote brain exposure, efficacy, and possible future systemic application.

Keywords: ASO; blood brain barrier; drug discovery; oligonucleotide; siRNA.

Publication types

Review

Abstract

Publication types

Grants and funding