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

• Main Authors: Xiang Li (Author), Mahboubeh Kheirabadi (Author), Patrick G. Dougherty (Author), Kimberli J. Kamer (Author), Xiulong Shen (Author), Nelsa L. Estrella (Author), Suresh Peddigari (Author), Anushree Pathak (Author), Sara L. Blake (Author), Emmanuelle Sizensky (Author), Carmen del Genio (Author), Arti B. Gaur (Author), Mohanraj Dhanabal (Author), Mahasweta Girgenrath (Author), Natarajan Sethuraman (Author), Ziqing Qian (Author)
• Format: Book
• Published: Elsevier, 2023-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...