File:1-s2.0-S1525001621001957-gr1.jpg

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Description
English: Figure 1. Summary of gene therapy strategies (A) Non-viral strategies include using ASOs to induce alternate splicing or RNase H-mediated degradation. (B and C) Viral strategies include (B) AAV-mediated gene silencing, through RNA interference or CRISPR-Cas9 or (C) AAV-mediated gene delivery including neurotrophic factors. AAV, adeno-associated virus; ASO, antisense oligonucleotide; Cas, CRISPR-associated system; miRNA, microRNA; PAM, protospacer adjacent motif; RISC, RNA-induced silencing complex; RNAi, RNA interference; shRNA, small hairpin RNA.
Date
Source https://www.sciencedirect.com/science/article/pii/S1525001621001957
Author Defne A. Amado , Beverly L. Davidson

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