Liposomal formulations for treating lysosomal storage disorders.
Adv Drug Deliv Rev
; 190: 114531, 2022 11.
Article
in En
| MEDLINE
| ID: mdl-36089182
ABSTRACT
Lysosomal storage disorders (LSD) are a group of rare life-threatening diseases caused by a lysosomal dysfunction, usually due to the lack of a single enzyme required for the metabolism of macromolecules, which leads to a lysosomal accumulation of specific substrates, resulting in severe disease manifestations and early death. There is currently no definitive cure for LSD, and despite the approval of certain therapies, their effectiveness is limited. Therefore, an appropriate nanocarrier could help improve the efficacy of some of these therapies. Liposomes show excellent properties as drug carriers, because they can entrap active therapeutic compounds offering protection, biocompatibility, and selectivity. Here, we discuss the potential of liposomes for LSD treatment and conduct a detailed analysis of promising liposomal formulations still in the preclinical development stage from various perspectives, including treatment strategy, manufacturing, characterization, and future directions for implementing liposomal formulations for LSD.
Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Lysosomal Storage Diseases
/
Liposomes
Limits:
Humans
Language:
En
Journal:
Adv Drug Deliv Rev
Journal subject:
FARMACOLOGIA
/
TERAPIA POR MEDICAMENTOS
Year:
2022
Type:
Article
Affiliation country:
Spain