Table 3.
Vesicle-based delivery systems of therapeutic molecules for the treatment of LSDs.
| Vesicle type | Modification | Carried enzymatic or other cargo | Condition associated with enzyme deficiency | Type of study | References |
|---|---|---|---|---|---|
| EXOSOMES | |||||
| HEK293-derived exosomes | Vesicular stomatitis virus glycoprotein for the improved le of carried cargo protein | β-glucocerebrosidase | Gaucher disease | In vitro study | Do et al., 2019 |
| macrophage-derived EVs | tripeptidyl peptidase-1 | Neuronal Ceroid Lipofuscinoses (NCL)-2, Batten disease | In vitro and In vivo study | Haney et al., 2019 | |
| LIPOSOMES | |||||
| liposome | β-galactosidase | GM1 gangliosidosis | In vivo study | Gregoriadis, 1978 | |
| liposome | α-mannosidase | Mannosidosis | In vivo study | Patel and Ryman, 1974 | |
| liposome | neuraminidase | Mucolipidosis type I | In vivo study | Gregoriadis et al., 1974 | |
| liposome | β-galactosidase | Krabbe disease | In vivo study | Umezawa et al., 1985 | |
| nanoliposomes | Functionalized with Arginine-Glycine-Aspartic acid (RGD) peptides for improved cellular uptake | α-galactosidase | Fabry disease | In vivo study | Cabrera et al., 2016 |
| GNeo-liposomes | Guanidinylated neomycin (GNeo) transporter for enhanced delivery to lysososmes | α-l-iduronidase | Mucopolysaccharidosis type I | In vitro study | Hamill et al., 2017 |
| liposomes | Lysosomotropic octadecyl-rhodamine B (Rh) for enhanced delivery to lysososmes | Glucocerebroside velaglucerase alfa | Gaucher disease | In vitro study | Koshkaryev et al., 2011 |
| NANOPARTICLES | |||||
| PLGA acidic NPs | Acidic NPs for re-acidification of the defective lysosomes | glucocerebrosidase-mutant cells, PD | In vitro and in vivo study | Bourdenx et al., 2016 | |
| NPs | saposin C for the protection of biologically active cargo | acid b-glucosidase | Pompe disease | In vivo study | Sun et al., 2020 |
| PLGA NPs | Ang2- (Angiopep-2), g7, and Tf2 (transferrin binding)-functionalized NPs to improve the delivery to brain | galactosylceramidase | Krabbe disease | In vivo study | Del Grosso et al., 2019 |
| polystyrene particle | Coupled to anti-ICAM-1 antibody for an improved targeting to organs | α-galactosidase | Fabry disease | In vivo and in vitro study | Hsu et al., 2014, Hsu et al., 2011 |
| polystyrene particle | Coupled to anti-ICAM-1 antibody for an improved targeting to organs | acid α-glucosidase | Pompe disease | In vivo study | Hsu et al., 2012 |
| NPs | Coupled to anti-ICAM-1 antibody for an improved targeting to organs | acid sphingomyelinase | Acid sphingomyelinase deficiency | In vitro study | Muro et al., 2006 |
| PLGA NPs | 7-aminoacid glycopeptide for increased targeting of NPs | Albumin was used as a model | NPs were tested in MPS-I and II models | In vivo and in vitro study | Salvalaio et al., 2016 |
| Protein-based NPs | Human serum albumin (HSA) and 30 Kc19 silkworm proteins were used to make the NPs to enhance cellular uptake | α-galactosidase | Fabry disease | In vitro study | Lee et al., 2016 |
| trimethyl chitosan-based polyelectrolyte complex-based nanocarriers | respond to low pH by the triggered release of the loaded protein for controlled release, Atto 647 N modification | α -galactosidase | Fabry disease | In vitro study | Giannotti et al., 2011 |
| quantum dots | Guanidinylated neomycin (GNeo) for improved cell binding | β-glucuronidase or α-iduronidase | MPS-VII and MPS-I | In vitro study | Sarrazin et al., 2010 |
ICAM-1, intercellular Adhesion Molecule 1; MPS, mucopolysaccharidosis; PD, Parkinson disease.