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An Injectable Thermoreversible Hydrogel Loaded with ManNAc for GNE Myopathy

2025-06-30Comunicação em conferência Acesso restrito
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datacite.subject.fosCiências Naturais::Ciências Biológicas
datacite.subject.fosEngenharia e Tecnologia::Engenharia dos Materiais
datacite.subject.fosCiências Médicas::Ciências da Saúde
datacite.subject.sdg03:Saúde de Qualidade
datacite.subject.sdg11:Cidades e Comunidades Sustentáveis
dc.contributor.authorViolante, Cristiana
dc.contributor.authorMarques-da-Silva, Dorinda
dc.contributor.authorVideira, Paula Alexandra
dc.contributor.authorLagoa, Ricardo
dc.date.accessioned2026-10-06T20:28:25Z
dc.date.available2026-10-06T20:28:25Z
dc.date.issued2025-06-30
dc.descriptionConference name: 6th International Conference on Bio-Engineering for Smart Technologies, BioSMART 2025 14 -16 May 2025
dc.descriptionAcknowledgment: 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).
dc.description.abstractGNE 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.eng
dc.description.sponsorshipThis 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).
dc.identifier.citationC. 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.
dc.identifier.doi10.1109/biosmart66413.2025.11046070
dc.identifier.isbn978-1-6654-7811-3
dc.identifier.issn2831-4352
dc.identifier.urihttp://hdl.handle.net/10400.8/16974
dc.language.isoeng
dc.peerreviewedyes
dc.publisherIEEE
dc.relationLaboratory of Separation and Reaction Engineering - Laboratory of Catalysis and Materials
dc.relationLaboratory of Separation and Reaction Engineering - Laboratory of Catalysis and Materials
dc.relationInstitute for Health and Bioeconomy
dc.relation.hasversionhttps://ieeexplore.ieee.org/document/11046070/
dc.relation.ispartof2025 6th International Conference on Bio-engineering for Smart Technologies (BioSMART)
dc.rights.uriN/A
dc.subjectBiomaterials
dc.subjectCongenital Disorders of Glycosylation
dc.subjectDrug delivery
dc.subjectGNE myopathy
dc.subjectHereditary inclusion body myopathy type 2
dc.subjectN-Acetylmannosamine
dc.subjectPluronic-F127
dc.titleAn Injectable Thermoreversible Hydrogel Loaded with ManNAc for GNE Myopathyeng
dc.title.alternative6th International Conference on Bio-Engineering for Smart Technologies, BioSMART 2025eng
dc.typeconference paper
dspace.entity.typePublication
oaire.awardNumberUIDB/50020/2020
oaire.awardNumberUIDP/50020/2020
oaire.awardNumberLA/P/0140/2020
oaire.awardTitleLaboratory of Separation and Reaction Engineering - Laboratory of Catalysis and Materials
oaire.awardTitleLaboratory of Separation and Reaction Engineering - Laboratory of Catalysis and Materials
oaire.awardTitleInstitute for Health and Bioeconomy
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/6817 - DCRRNI ID/UIDB%2F50020%2F2020/PT
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/6817 - DCRRNI ID/UIDP%2F50020%2F2020/PT
oaire.awardURIhttp://hdl.handle.net/10400.8/13764
oaire.citation.conferenceDate2025-05-14
oaire.citation.conferencePlaceParis, França
oaire.citation.title6th International Conference on Bio-engineering for Smart Technologies (BioSMART 2025)
oaire.fundingStream6817 - DCRRNI ID
oaire.fundingStream6817 - DCRRNI ID
oaire.fundingStream6817 - DCRRNI ID
oaire.versionhttp://purl.org/coar/version/c_970fb48d4fbd8a85
person.familyNameViolante
person.familyNameMarques da Silva
person.familyNameLagoa
person.givenNameCristiana
person.givenNameDorinda
person.givenNameRicardo
person.identifier124357
person.identifier.ciencia-id521A-35C1-078D
person.identifier.ciencia-id5C18-A29A-44AB
person.identifier.orcid0009-0003-6081-0427
person.identifier.orcid0000-0003-0460-5421
person.identifier.orcid0000-0003-2375-6612
person.identifier.scopus-author-id23051352300
project.funder.identifierhttp://doi.org/10.13039/501100001871
project.funder.identifierhttp://doi.org/10.13039/501100001871
project.funder.nameFundação para a Ciência e a Tecnologia
project.funder.nameFundação para a Ciência e a Tecnologia
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