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Oligonucleotides (ONs) are therapeutic macromolecules with great potential for the treatment of neurological conditions, including spinal muscular atrophy (SMA), a neurodegenerative disease. However, the neurovascular unit severely limits their distribution to the neural parenchyma of the brain and the spinal cord. Cell-penetrating peptides (CPPs) can be conjugated to oligonucleotides to increase their delivery across biological barriers. In this chapter, we describe the synthesis and conjugation of CPPs to oligonucleotides, and the use of a severe SMA mouse model to test in vivo the efficacy of CPP-delivered oligonucleotides, using ELISA, western blot, and TaqMan™ RT-qPCR assays.

Original publication





Publication Date





491 - 513


Antisense oligonucleotides, Cell-penetrating peptide, In vivo pup administrations, Peptide oligonucleotide conjugates, Spinal muscular atrophy, Survival motor neuron, Taiwanese SMA mouse model, Animals, Cell-Penetrating Peptides, Disease Models, Animal, Mice, Muscular Atrophy, Spinal, Oligonucleotides, Oligonucleotides, Antisense