Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy

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...

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Những tác giả chính:	Shabanpoor, F, Hammond, S, Abendroth, F, Hazell, G, Wood, M, Gait, M
Định dạng:	Journal article
Ngôn ngữ:	English
Được phát hành:	Mary Ann Liebert 2017

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