| Literature DB >> 34766309 |
Larissa Goli1,2, Jessica Stoodley1, Suzan M Hammond1, Richard Raz3.
Abstract
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.Entities:
Keywords: Antisense oligonucleotides; Cell-penetrating peptide; In vivo pup administrations; Peptide oligonucleotide conjugates; Spinal muscular atrophy; Survival motor neuron; Taiwanese SMA mouse model
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Year: 2022 PMID： 34766309 DOI： 10.1007/978-1-0716-1752-6_31
Source DB: PubMed Journal: Methods Mol Biol ISSN： 1064-3745