Distribution and Penetration of Intracerebroventricularly Administered 2′OMePS Oligonucleotide in the Mouse Brain
作者： João Casaca-Carreira , Yasin Temel , Iñaki Larrakoetxea , Ali Jahanshahi
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摘要: Antisense oligonucleotide (AON) therapy is emerging as a potential treatment strategy for neurodegenerative diseases, such spinal muscular atrophy, Huntington's disease, and amyotrophic lateral sclerosis. AONs function at the cellular level by, example, direct interference with expression of gene products or molecular activation neuroprotective pathways. However, AON faces major obstacle limiting its clinical application central nervous system (CNS) disorders: blood–brain barrier. Systemic administration leads to rapid clearance breakdown molecules in periphery. One way overcome this intracerebroventricular (ICV) delivery therapeutics directly cerebrospinal fluid (CSF). Given particular structure oligonucleotides, (pharmaco) kinetic distribution pattern these compounds brain are yet be clarified. In study, 2′OMePS delivered through ICV into CSF reached most key...
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