TABLE 2.
List of published zebrafish LSD models.
| Gene§ | LSD | Zebrafish model | References |
| Sphingolipidoses | |||
| ASAH1* | Farber lipogranulomatosis | Morpholino (asah1b) | Zhou et al., 2012 |
| asah1a+68/+68; asah1b–20/–20 (CRISPR-Cas9) | Zhang T. et al., 2019 | ||
| ARSA | Metachromatic leukodystrophy | Morpholino | Berg et al., 2016 |
| GALC* | Globoid cell leukodystrophy/Krabbe disease | Morpholino (galca) | Zizioli et al., 2014 |
| Morpholino (galcb) | Zizioli et al., 2014 | ||
| Morpholino (galca; galcb) | Zizioli et al., 2014 | ||
| GBA | Gaucher disease | Morpholino | Zancan et al., 2014 |
| gbasa1621/sa1621 (ENU) | Busch-Nentwich et al., 2012; Zancan et al., 2014 | ||
| gbasa391/sa391 (TALENs) | Keatinge et al., 2015; Watson et al., 2019 | ||
| Morpholino | Berg et al., 2016 | ||
| gbaΔ31/Δ31 (CRISPR-Cas9) | Lelieveld et al., 2019 | ||
| HEXA | Tay-Sachs disease | Morpholino | Berg et al., 2016 |
| HEXB | Sandhoff disease | Morpholino | Kalén et al., 2009 |
| herb–14/–14 (CRISPR-Cas9) | Kuil et al., 2019 | ||
| Mucolipidoses | |||
| GNPTAB | Mucolipidosis II α/β (I-cell disease); mucolipidosis III α/β (pseudo-Hurler polydystrophy) | Morpholino | Flanagan-Steet et al., 2009, 2016a, 2018; Petrey et al., 2012; Qian et al., 2015 |
| gnptab–/– Line 1 (TALENs) | Flanagan-Steet et al., 2018 | ||
| gnptab–/– Line 2 (TALENs) | Flanagan-Steet et al., 2018 | ||
| GNPTG | Mucolipidosis III γ, variant pseudo-Hurler polydystrophy | gnptgzm00105646Tg/zm00105646Tg (DNA insertion) | Flanagan-Steet et al., 2016b |
| MCOLN1* | Mucolipidosis IV | mcoln1ahg82/hg82; mcoln1bhg84/hg84 (TALENs/CRISPR-Cas9) | Li et al., 2017 |
| mcoln1ahg82/hg82 (TALENs) | Li et al., 2017 | ||
| mcoln1bhg84/hg84 (CRISPR-Cas9) | Li et al., 2017 | ||
| mcoln1ahkz13/hkz13 (biluo; ENU) | Jin et al., 2019 | ||
| Neuronal ceroid lipofuscinoses | |||
| ATP13A2 | CLN12/Kufor-Rakeb syndrome | Morpholino | Lopes da Fonseca et al., 2013 |
| Morpholino | Spataro et al., 2019 | ||
| CLN3 | CLN3/Batten-Spielmeyer-Sjogren disease | Morpholino | Wager et al., 2016 |
| Morpholino | Wager et al., 2016 | ||
| CTSD | CLN10 | Morpholino | Follo et al., 2011; Follo et al., 2013 |
| GRN* | CLN11 | Morpholino (grna) | Li et al., 2010, 2013 |
| Morpholino (grnb) | Li et al., 2013 | ||
| Morpholinos (2 grna lines) | Chitramuthu et al., 2010, 2017b | ||
| Morpholino (grnb) | Chitramuthu et al., 2010 | ||
| Morpholinos (2 grna lines, 2 grnb lines, 1 grna; grnb line) | Laird et al., 2010; De Muynck et al., 2013 | ||
| TPP1 | CLN2 | tpp1sa11/sa11 (ENU) | Busch-Nentwich et al., 2010b; Busch-Nentwich et al., 2013; Mahmood et al., 2013 |
| tpp1hu3587/hu3587 (ENU) | Busch-Nentwich et al., 2010a; Mahmood et al., 2013 | ||
| Morpholinos (2 lines) | Mahmood et al., 2013 | ||
| Integral membrane protein disorders | |||
| CTNS | Cystinosis | ctnssa14661/sa14661 (ENU) | Busch-Nentwich et al., 2013; Elmonem et al., 2017 |
| Morpholino | Elmonem et al., 2017 | ||
| ctnszh601/zh601 (TALENs) | Festa et al., 2018 | ||
| NPC1 | Niemann-Pick disease type C1; type D | Morpholinos (2 lines) | Schwend et al., 2011; Louwette et al., 2012; Chu et al., 2015 |
| npc1ihb334/ihb334 (CRISPR-Cas9) | Lin et al., 2018 | ||
| npc1ihb335/ihb335 (CRISPR-Cas9) | Lin et al., 2018 | ||
| npc1hg37/hg37 (CRISPR-Cas9) | Tseng et al., 2018 | ||
| npc1y535/y535 (CRISPR-Cas9) | Tseng et al., 2018 | ||
| SCARB2* | Action myoclonus-renal syndrome | scarb2ahi1463Tg/hi1463Tg (DNA insertion) | Golling et al., 2002; Amsterdam et al., 2004; Diaz-Tellez et al., 2016 |
| scarb2ahi2715Tg/hi2715Tg (DNA insertion) | Amsterdam et al., 2004 | ||
| Glycogen storage disease | |||
| GAA | Pompe disease | gaazf752/zf752 (TALENs) | Wu et al., 2017 |
| Glycoproteinosis | |||
| MANBA | β-mannosidosis | Morpholino | Ko et al., 2017 |
| Mucopolysaccharidosis | |||
| IDS | Mucopolysaccharidosis II/Hunter syndrome | Morpholino | Moro et al., 2010; Costa et al., 2017; Bellesso et al., 2018 |
| idsia200/ia200 (CRISPR-Cas9) | Bellesso et al., 2018 | ||
| Lysosome-related organelle (LRO) disorders | |||
| HSP5 | Hermansky-Pudlak disease type 5 | hsp5m454/m454 (snow white; ENU) | Driever et al., 1996; Stemple et al., 1996; Daly et al., 2013 |
| Morpholino | Daly et al., 2013 | ||
| HSP7* | Hermansky-Pudlak disease type 7 | dtnbp1aihb819/ihb819 (CRISPR-Cas9) | Chen et al., 2018 |
| LYST | Chédiak-Higashi disease | lystmuz107/muz107 (ENU) | Kim et al., 2015 |
§Gene denotes human gene names. *Human genes with more than one known orthologue in the zebrafish, the orthologue(s) targeted for mutation are denoted in the names of the zebrafish models.