Towards Personalized Allele-Specific Antisense Oligonucleotide Therapies for Toxic Gain-of-Function Neurodegenerative Diseases

Towards Personalized Allele-Specific Antisense Oligonucleotide Therapies for Toxic Gain-of-Function Neurodegenerative Diseases

Antisense oligonucleotides (ASOs) are single-stranded nucleic acid strings that can be used to selectively modify protein synthesis by binding complementary (pre-)mRNA sequences. By specific arrangements of DNA and RNA into a chain of nucleic acids and additional modifications of the backbone, sugar...

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Bibliographic Details
Main Authors: Jacob Helm, Ludger Schöls, Stefan Hauser
Format: Article
Language:English
Published: MDPI AG 2022-08-01
Series:Pharmaceutics
Subjects:
antisense oligonucleotide
ASO
neurodegenerative diseases
toxic gain-of-function
allele-specific targeting
SNP
Online Access:https://www.mdpi.com/1999-4923/14/8/1708

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https://www.mdpi.com/1999-4923/14/8/1708

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