期刊
HUMAN MOLECULAR GENETICS
卷 20, 期 -, 页码 R21-R27出版社
OXFORD UNIV PRESS
DOI: 10.1093/hmg/ddr137
关键词
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资金
- National Institutes of Health [NS050210, NS067111, HD44093]
- Hereditary Disease Foundation
- Roy J. Carver Trust
RNAi interference (RNAi) is a powerful gene silencing technology that has immense potential for treating a vast array of human ailments, for which suppressing disease-associated genes may provide clinical benefit. Here, we review the development of RNAi as a therapeutic modality for neurodegenerative diseases affecting the central nervous system (CNS). We overview promising preclinical data for the application of RNAi in the CNS and discuss key challenges (e. g. delivery and specificity) that remain as these approaches transition to the clinic.
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