Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy
Splice-switching antisense oligonucleotides are emerging treatments for neuromuscular diseases, with several splice-switching oligonucleotides (SSOs) currently undergoing clinical trials such as for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). However, the development of syst...
Main Authors: | Shabanpoor, F, Hammond, S, Abendroth, F, Hazell, G, Wood, M, Gait, M |
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פורמט: | Journal article |
שפה: | English |
יצא לאור: |
Mary Ann Liebert
2017
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