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Identifying and Avoiding tcDNA-ASO Sequence-Specific Toxicity for the Development of DMD Exon 51 Skipping Therapy

Tricyclo-DNA (tcDNA) antisense oligonucleotides (ASOs) hold promise for therapeutic splice-switching applications and the treatment of Duchenne muscular dystrophy (DMD) in particular. We have previously reported the therapeutic potential of tcDNA-ASO in mouse models of DMD, highlighting their unique...

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Detalles Bibliográficos
Autores principales:	Aupy, Philippine, Echevarría, Lucía, Relizani, Karima, Zarrouki, Faouzi, Haeberli, Adrian, Komisarski, Marek, Tensorer, Thomas, Jouvion, Grégory, Svinartchouk, Fedor, Garcia, Luis, Goyenvalle, Aurélie
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7063478/ https://www.ncbi.nlm.nih.gov/pubmed/31881528 http://dx.doi.org/10.1016/j.omtn.2019.11.020

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