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dc.contributorUniversitat Ramon Llull. IQS
dc.contributor.authorGuerra Rebollo, Marta
dc.contributor.authorStampa, María
dc.contributor.authorLázaro, Miguel Ángel
dc.contributor.authorCascante, Anna
dc.contributor.authorFornaguera Puigvert, Cristina
dc.contributor.authorBorrós i Gómez, Salvador
dc.date.accessioned2024-07-09T15:30:15Z
dc.date.available2024-07-09T15:30:15Z
dc.date.issued2021-09-14
dc.identifier.issn2214-3602ca
dc.identifier.urihttp://hdl.handle.net/20.500.14342/4155
dc.description.abstractBackground:Duchenne Muscular Dystrophy (DMD) is one of the most common muscular dystrophies, caused by mutated forms of the dystrophin gene. Currently, the only treatment available is symptoms management. Novel approximations are trying to treat these patients with gene therapy, namely, using viral vectors. However, these vectors can be recognized by the immune system decreasing their therapeutic activity and making impossible a multidose treatment due to the induction of the humoral immunity following the first dose. Objective:Our objective is to demonstrate the feasibility of using a hybrid vector to avoid immune clearance, based on the electrostatic coating of adeno-associated virus (AAVs) vectors with our proprietary polymers. Methods:We coated model adeno-associated virus vectors by electrostatic interaction of our cationic poly (beta aminoester) polymers with the viral anionic capsid and characterized biophysical properties. Once the nanoformulations were designed, we studied their in vivo biodistribution by bioluminescence analysis and we finally studied the capacity of the polymers as potential coatings to avoid antibody neutralization. Results:We tested two polymer combinations and we demonstrated the need for poly(ethylene glycol) addition to avoid vector aggregation after coating. In vivo biodistribution studies demonstrated that viral particles are located in the liver (short times) and also in muscles (long times), the target organ. However, we did not achieve complete antibody neutralization shielding using this electrostatic coating. Conclusions:The null hypothesis stands: although it is feasible to coat viral particles by electrostatic interaction with a proprietary polymer, this strategy is not appropriate for AAVs due to their small size, so other alternatives are required as a novel treatment for DMD patients.ca
dc.format.extent30 p.ca
dc.language.isoengca
dc.publisherIOS Pressca
dc.relation.ispartofJournal of Neuromuscular Diseases, vol. 8, no. 5, pp. 815-825ca
dc.rights© L'autor/aca
dc.rightsAttribution 4.0 Internationalca
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/*
dc.subject.otherpBAE nanoparticlesca
dc.subject.otherAdeno-associated virusesca
dc.subject.otherViral coatingca
dc.subject.otherDuchenne Muscular Dystrophyca
dc.subject.otherAntibodyca
dc.subject.otherNeutralizationca
dc.subject.otherHybrid vectorsca
dc.subject.otherBiodistribution studiesca
dc.subject.otherNanopartículesca
dc.subject.otherAdenovirusca
dc.subject.otherDistròfia muscular de Duchenneca
dc.subject.otherImmunogenèticaca
dc.titleElectrostatic Coating of Viral Particles for Gene Delivery Applications in Muscular Dystrophies: Influence of Size on Stability and Antibody Protectionca
dc.typeinfo:eu-repo/semantics/articleca
dc.rights.accessLevelinfo:eu-repo/semantics/openAccess
dc.rights.accessLevelinfo:eu-repo/semantics/openAccess
dc.embargo.termscapca
dc.subject.udc616.7ca
dc.identifier.doihttps://doi.org/10.3233/JND-210662ca
dc.description.versioninfo:eu-repo/semantics/acceptedVersionca


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