Nucleic Acid–Based Therapeutics in Orphan Neurological Disorders: Recent Developments

Nucleic Acid–Based Therapeutics in Orphan Neurological Disorders: Recent Developments

The possibility of rational design and the resulting faster and more cost-efficient development cycles of nucleic acid–based therapeutics (NBTs), such as antisense oligonucleotides, siRNAs, and gene therapy vectors, have fueled increased activity in developing therapies for orphan diseases. Despite...

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Bibliographic Details
Main Authors: Olga Khorkova, Jane Hsiao, Claes Wahlestedt
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-04-01
Series:Frontiers in Molecular Biosciences
Subjects:
orphan disorder
neurological disorder
antisense oligonucleotide
siRNA
gene therapy
noncoding RNA
Online Access:https://www.frontiersin.org/articles/10.3389/fmolb.2021.643681/full

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