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. 2018 Jan 15;6(1):9. doi: 10.3390/biomedicines6010009

Table 1.

Main RNA-therapy studies in amyotrophic lateral sclerosis.

Year Reference Organism Gene Target Therapeutic Strategy
Animal Human Cells Human SOD1 C9ORF72 FUS ATXN2 ASOs SMT GT
2003 Ding et al. [8] D + +
2005 Miller et al. [9] M + +
2005 Ralph et al. [10] M + +
2006 Smith et al. [11] R/NHP + +
2013 Donnelly et al. [12] + + +
2013 Foust et al. [13] M + +
2013 Koval et al. [14] M + +
2013 Lagier-Tourenne et al. [15] M + + +
2013 Miller et al. [16] + + +
2013 Sareen et al. [17] + + +
2015 Butovsky et al. [18] M + +
2016 Borel et al. [19] M/NHP + +
2016 Jiang et al. [20] M + +
2017 Becker et al. [21] M + +
2017 Biferi et al. [22] M + +
2017 Wang et al. [23] + + +
2017 Chen et al. [24] Z + +

ASOs: antisense oligonucleotides; D: drosophila; GT: gene therapy; M: mouse; NHP: nonhuman primate; R: rat; SMT: small molecule therapy; Z: zebrafish.