An Injectable Thermoreversible Hydrogel Loaded with ManNAc for GNE Myopathy

Violante, Cristiana; Marques-da-Silva, Dorinda; Videira, Paula Alexandra; Lagoa, Ricardo

http://hdl.handle.net/10400.8/16974

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Autores

Violante, Cristiana

Marques-da-Silva, Dorinda

Videira, Paula Alexandra

Lagoa, Ricardo

Resumo(s)

GNE myopathy (GNEM) is a rare adult-onset disease characterized by atrophy and weakness of skeletal muscles caused by mutations in the GNE gene. There is no approved therapy, but clinical trials with supplementation with N-acetylmannosamine (ManNAc) alleviated the GNEM-associated decrease in sialylation. Nevertheless, oral intake of ManNAc has shown low absorption and high gastrointestinal adverse effects. In this regard, the aim of this work was to explore an alternative delivery route for ManNAc directly targeting the muscle tissues. For this we investigated the thermal responses of Pluronic F-127 gel and its muscle adhesion after subcutaneous injection, and the kinetics of release of gelencapsulated ManNAc in phosphate buffered saline medium. The results obtained show the thermal response of Pluronic F-127 represents a unique opportunity for its injectability and function as a drug delivery system directly acting on the muscle surrounding area and showing gel stability in the target area. Moreover, the continuous delivery of ManNAc from the gel (20% Pluronic F-127) was detected during 3h, at a continuous rate and slower than using a simple ManNAc solution. In conclusion, the data obtained in this work supports the possible use of the Pluronic F-127 thermoreversible hydrogel loaded with ManNAc in GNE myopathy therapy and research.

Descrição

Conference name: 6th International Conference on Bio-Engineering for Smart Technologies, BioSMART 2025 14 -16 May 2025
Acknowledgment: This research was funded by FCT/MCTES (PIDDAC, Portugal) under the programmatic projects UIDB/50020/2020, UIDP/50020/2020 and LA/P/0140/2020; and by the European Commission through GLYCOTwinning (Grant Agreement: 101079417), and Horizon 2020 research and innovation programme under the EJPRD COFUND-EJP N 825575 (EJPRD/0001/2020).

Palavras-chave

Biomaterials Congenital Disorders of Glycosylation Drug delivery GNE myopathy Hereditary inclusion body myopathy type 2 N-Acetylmannosamine Pluronic-F127

URI

http://hdl.handle.net/10400.8/16974

Citação

C. Violante, D. Marques-Da-Silva, P. A. Videira and R. Lagoa, "An Injectable Thermoreversible Hydrogel Loaded with ManNAc for GNE Myopathy," 2025 6th International Conference on Bio-engineering for Smart Technologies (BioSMART), Paris, France, 2025, pp. 1-4, doi: 10.1109/BioSMART66413.2025.11046070.