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Nucleic Acid–Based Therapeutics in Orphan Neurological Disorders: Recent Developments

The possibility of rational design and the resulting faster and more cost-efficient development cycles of nucleic acid–based therapeutics (NBTs), such as antisense oligonucleotides, siRNAs, and gene therapy vectors, have fueled increased activity in developing therapies for orphan diseases. Despite...

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Detalles Bibliográficos
Autores principales:	Khorkova, Olga, Hsiao, Jane, Wahlestedt, Claes
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2021
Materias:	Molecular Biosciences
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8115123/ https://www.ncbi.nlm.nih.gov/pubmed/33996898 http://dx.doi.org/10.3389/fmolb.2021.643681

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