Table 1.

Particular examples of translational successes using the zebrafish model for drug discovery

Human disease	Zebrafish model	Outcome	References
Pontocerebellar hyperplasia	Tsen54 antisense morpholino	Linking a loss‐of‐function mutation in the tsen54 gene to brain hypoplasia	(Kasher et al., 2011)
	R44X‐loss‐of‐function mutant	Linking homozygous mutation of CLP1 (a member of the tRNA splicing endonuclease complex, TSEN) to abnormal spinal neurons, curved body, small head and eyes, and an early death in fish helped identify this mutation as a risk factor for human conditions	(Schaffer et al., 2014)
Spinal cord injury	Heat shock transgenic lines	Zebrafish show high capacity for axonal regeneration following spinal cord injury, especially through the activation of Fgf signalling. Increasing Fgf signalling in mammalian spinal injury sites may encourage glial cell differentiation and lead to favourable conditions for axonal regeneration	(Goldshmit et al., 2012)
Schizophrenia	Tg(huC:eGFP)	The Rgs4 gene is associated with the onset and development of schizophrenia. Using the transgenic zebrafish line, rgs4 was found to be essential for axon formation, providing the first in vivo evidence supporting the role of rgs4 in schizophrenia	(Cheng et al., 2013)