Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment

Spinal muscular atrophy (SMA) is a recessive disease caused by mutations in the SMN1 gene, which encodes the protein survival motor neuron (SMN), whose absence dramatically affects the survival of motor neurons. In humans, the severity of the disease is lessened by the presence of a gene copy, SMN2....

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Detalles Bibliográficos
Autores principales: Robin, Valérie, Griffith, Graziella, Carter, John-Paul L., Leumann, Christian J., 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/PMC5415958/
https://www.ncbi.nlm.nih.gov/pubmed/28624227
http://dx.doi.org/10.1016/j.omtn.2017.02.009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5415958/
https://www.ncbi.nlm.nih.gov/pubmed/28624227
http://dx.doi.org/10.1016/j.omtn.2017.02.009

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