The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders

Biological therapeutic agents are highly targeted and potent but limited in their ability to reach intracellular targets. These limitations often necessitate high therapeutic doses and can be associated with less-than-optimal therapeutic activity. One promising solution for therapeutic agent deliver...

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Detalles Bibliográficos
Autores principales: Li, Xiang, Kheirabadi, Mahboubeh, Dougherty, Patrick G., Kamer, Kimberli J., Shen, Xiulong, Estrella, Nelsa L., Peddigari, Suresh, Pathak, Anushree, Blake, Sara L., Sizensky, Emmanuelle, Genio, Carmen del, Gaur, Arti B., Dhanabal, Mohanraj, Girgenrath, Mahasweta, Sethuraman, Natarajan, Qian, Ziqing
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10393622/
https://www.ncbi.nlm.nih.gov/pubmed/37538053
http://dx.doi.org/10.1016/j.omtn.2023.06.022

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10393622/
https://www.ncbi.nlm.nih.gov/pubmed/37538053
http://dx.doi.org/10.1016/j.omtn.2023.06.022

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