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.