Article
Efficient Gene Suppression in Dorsal Root Ganglia and Spinal Cord Using Adeno-Associated Virus Vectors Encoding Short-Hairpin RNA.
Methods in molecular biology (Clifton, N.J.) - 1 Jan 2016
Enomoto Mitsuhiro, Hirai Takashi, Kaburagi Hidetoshi, Yokota Takanori
Abstract excerpt
RNA interference is a powerful tool used to induce loss-of-function phenotypes through post-transcriptional gene silencing. Small interfering RNA (siRNA) molecules have been used to target the central nervous system (CNS) and are expected to have clinical utility against refractory neurodegenerative diseases. However, siRNA is characterized by low transduction efficiency, insufficient inhibition of gene...
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