| Autor: |
Noelle D, Germain, Wendy K, Chung, Patrick D, Sarmiere |
| Rok vydání: |
2023 |
| Předmět: |
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| Zdroj: |
Molecular Aspects of Medicine. 91:101148 |
| ISSN: |
0098-2997 |
| DOI: |
10.1016/j.mam.2022.101148 |
| Popis: |
Advances in genome sequencing have greatly facilitated the identification of genomic variants underlying rare neurodevelopmental and neurodegenerative disorders. Understanding the fundamental causes of rare monogenic disorders has made gene therapy a possible treatment approach for these conditions. RNA interference (RNAi) technologies such as small interfering RNA (siRNA), microRNA (miRNA), and short hairpin RNA (shRNA), and other oligonucleotide-based modalities such as antisense oligonucleotides (ASOs) are being developed as potential therapeutic approaches for manipulating expression of the genes that cause a variety of neurological diseases. Here, we offer a brief review of the mechanism of action of these RNAi approaches; provide deeper discussion of the advantages, challenges, and specific considerations related to the development of RNAi therapeutics for neurological disease; and highlight examples of rare neurological diseases for which RNAi therapeutics hold great promise. |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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Full Text from ScienceDirect