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.