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.
Bioactive materials for therapy and diagnosis
Liposomal formulations for treating lysosomal storage disorders
Judit Tomsen-Melero, Josep Merlo-Mas, Aida Carreño, Santi Sala, Alba Córdoba, Jaume Veciana, Elisabet González-Mira, Nora Ventosa
Advanced Drug Delivery Reviews Volume 190, November 2022, 114531