Evaluation of cell-penetrating peptide delivery of antisense oligonucleotides for therapeutic efficacy in spinal muscular atrophy

Evaluation of cell-penetrating peptide delivery of antisense oligonucleotides for therapeutic efficacy in spinal muscular atrophy

Antisense oligonucleotides (ASOs) are a widely used form of gene therapy, which is translatable to multiple disorders. A major obstacle for ASO efficacy is its bioavailability for in vivo and in vitro studies. To overcome this challenge we use cell-penetrating peptides (CPPs) for systemic delivery o...

書目詳細資料
Main Authors:	Hammond, S, Abendroth, F, Gait, M, Wood, M
格式:	Journal article
語言:	English
出版:	Springer 2019

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