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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Detalles Bibliográficos
Autores principales: Helm, Jacob, Schöls, Ludger, Hauser, Stefan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9416334/
https://www.ncbi.nlm.nih.gov/pubmed/36015334
http://dx.doi.org/10.3390/pharmaceutics14081708

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