Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice

• Egile Nagusiak: Mingxing Wang (Egilea), Bo Wu (Egilea), Sapana N Shah (Egilea), Peijuan Lu (Egilea), Qilong Lu (Egilea)
• Formatua: Liburua
• Argitaratua: Taylor & Francis Group, 2017-01-01T00:00:00Z.
• Gaiak: article
• Sarrera elektronikoa: Connect to this object online.

Antisense oligonucleotide therapy for Duchenne muscular dystrophy has shown great potential in preclinical and clinical trials, but its therapeutic applications are still limited due to inefficient delivery. In this study, we investigated a few polyquaterniums (PQs) with different size and compositi...