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Antisense Oligonucleotide Therapy for the Nervous System: From Bench to Bedside with Emphasis on Pediatric Neurology
Antisense oligonucleotides (ASOs) are disease-modifying agents affecting protein-coding and noncoding ribonucleic acids. Depending on the chemical modification and the location of hybridization, ASOs are able to reduce the level of toxic proteins, increase the level of functional protein, or modify...
Autores principales: | Amanat, Man, Nemeth, Christina L., Fine, Amena Smith, Leung, Doris G., Fatemi, Ali |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9695718/ https://www.ncbi.nlm.nih.gov/pubmed/36365206 http://dx.doi.org/10.3390/pharmaceutics14112389 |
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