What Can RNA-Based Therapy Do for Monogenic Diseases?

The use of RNA-based approaches to treat monogenic diseases (i.e., hereditary disorders caused by mutations in single genes) has been developed on different fronts. One approach uses small antisense oligonucleotides (ASOs) to modulate RNA processing at various stages; namely, to enhance correct spli...

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Detalles Bibliográficos
Autores principales: Clarke, Luka A., Amaral, Margarida D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9863139/
https://www.ncbi.nlm.nih.gov/pubmed/36678889
http://dx.doi.org/10.3390/pharmaceutics15010260

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9863139/
https://www.ncbi.nlm.nih.gov/pubmed/36678889
http://dx.doi.org/10.3390/pharmaceutics15010260

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