Table 3.
Summary of the studied treatment approaches in systematically reviewed studies
| Type of galactosemia | Treatment approach | Animal/cellular model | References |
|---|---|---|---|
| GALT deficiency | GALK inhibition | Saccharomyces cerevisiae | Ross et al33; Mumma et al46; De‐Souza et al64; Machado et al73 |
| Patient cell lines (fibroblasts) | Tang et al74 | ||
| Drosophila melanogaster | Jumbo‐Lucioni et al58; Daenzer et al34 | ||
| Antioxidants | Drosophila melanogaster | Jumbo‐Lucioni et al,66, 67 | |
| Pharmacological chaperones | Escherichia coli | Coelho et al75 | |
| Patient cell lines (fibroblasts) | Haskovic et al76 | ||
| Antisense therapy | Escherichia coli | Coelho et al77 | |
| ER stress reducers | Saccharomyces cerevisiae | De‐Souza et al64 | |
| Mouse | Balakrishnan et al60, 61 | ||
| UGP up‐regulation | Saccharomyces cerevisiae | Lai and Elsas78 | |
| Patient cell lines (SV40‐transformed fibroblasts) | Lai et al5 | ||
| Uridine supplementation | Patient cell lines (erythrocytes) | Ng et al6 | |
| GALE deficiency | GALK inhibition | Saccharomyces cerevisiae | Ross et al33; Mumma et al46 |
| Uridine supplementation | ldlD cells | Schulz et al69 |
Abbreviations: ER, endoplasmic reticulum; GALE, uridine diphosphate‐galactose 4′‐epimerase; GALK, galactokinase.