Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model

Antisense oligonucleotides (AONs) hold promise for therapeutic splice-switching correction in many genetic diseases. However, despite advances in AON chemistry and design, systemic use of AONs is limited due to poor tissue uptake and sufficient therapeutic efficacy is still difficult to achieve. A n...

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Detalles Bibliográficos
Autores principales: Relizani, Karima, Griffith, Graziella, Echevarría, Lucía, Zarrouki, Faouzi, Facchinetti, Patricia, Vaillend, Cyrille, Leumann, Christian, Garcia, Luis, Goyenvalle, Aurélie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5498286/
https://www.ncbi.nlm.nih.gov/pubmed/28918017
http://dx.doi.org/10.1016/j.omtn.2017.06.013

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