Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases
Abstract
Multiple neurodegenerative diseases are characterized by single-protein dysfunction and aggregation. Treatment strategies for these diseases have often targeted downstream pathways to ameliorate consequences of protein dysfunction; however, targeting the source of that dysfunction, the affected protein itself, seems most judicious to achieve a highly effective therapeutic outcome. Antisense oligonucleotides (ASOs) are small sequences of DNA able...
Paper Details
Title
Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases
Published Date
Jun 1, 2017
Journal
Volume
94
Issue
6
Pages
1056 - 1070
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