{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T01:10:10Z","timestamp":1777857010024,"version":"3.51.4"},"reference-count":32,"publisher":"SAGE Publications","issue":"5","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JND"],"published-print":{"date-parts":[[2021,9,14]]},"abstract":"<jats:p>Background: 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.<\/jats:p>","DOI":"10.3233\/jnd-210662","type":"journal-article","created":{"date-parts":[[2021,8,3]],"date-time":"2021-08-03T12:57:43Z","timestamp":1627995463000},"page":"815-825","source":"Crossref","is-referenced-by-count":3,"title":["Electrostatic Coating of Viral Particles for Gene Delivery Applications in Muscular Dystrophies: Influence of Size on Stability and Antibody Protection"],"prefix":"10.1177","volume":"8","author":[{"given":"Marta","family":"Guerra-Rebollo","sequence":"first","affiliation":[{"name":"Grup d\u2019Enginyeria de Materials (Gemat), Institut Qu\u00edmic de Sarri\u00e1 (IQS), Universitat Ramon Llull (URL), Via Augusta, Barcelona, Spain"},{"name":"Sagetis Biotech, Via Augusta, Barcelona, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mar\u00eda","family":"Stampa","sequence":"additional","affiliation":[{"name":"Grup d\u2019Enginyeria de Materials (Gemat), Institut Qu\u00edmic de Sarri\u00e1 (IQS), Universitat Ramon Llull (URL), Via Augusta, Barcelona, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Miguel \u00c1ngel","family":"L\u00e1zaro","sequence":"additional","affiliation":[{"name":"Sagetis Biotech, Via Augusta, Barcelona, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anna","family":"Cascante","sequence":"additional","affiliation":[{"name":"Grup d\u2019Enginyeria de Materials (Gemat), Institut Qu\u00edmic de Sarri\u00e1 (IQS), Universitat Ramon Llull (URL), Via Augusta, Barcelona, Spain"},{"name":"Sagetis Biotech, Via Augusta, Barcelona, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cristina","family":"Fornaguera","sequence":"additional","affiliation":[{"name":"Grup d\u2019Enginyeria de Materials (Gemat), Institut Qu\u00edmic de Sarri\u00e1 (IQS), Universitat Ramon Llull (URL), Via Augusta, Barcelona, Spain"},{"name":"Sagetis Biotech, Via Augusta, Barcelona, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Salvador","family":"Borr\u00f3s","sequence":"additional","affiliation":[{"name":"Grup d\u2019Enginyeria de Materials (Gemat), Institut Qu\u00edmic de Sarri\u00e1 (IQS), Universitat Ramon Llull (URL), Via Augusta, Barcelona, Spain"},{"name":"Sagetis Biotech, Via Augusta, Barcelona, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","reference":[{"issue":"3","key":"10.3233\/JND-210662_ref1","first-page":"1223","article-title":"The Dystrophin Complex: Structure, function and implications for therapy","volume":"5","author":"Gao","year":"2016","journal-title":"Compr Physiol"},{"issue":"3","key":"10.3233\/JND-210662_ref2","doi-asserted-by":"crossref","first-page":"509","DOI":"10.1016\/0092-8674(87)90504-6","article-title":"Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals","volume":"50","author":"Koenig","year":"1987","journal-title":"Cell"},{"issue":"4","key":"10.3233\/JND-210662_ref3","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1002\/cpmo.58","article-title":"Viral Vectors for Gene Transfer","volume":"8","author":"Chen","year":"2018","journal-title":"Curr Protoc Mouse Biol"},{"key":"10.3233\/JND-210662_ref4","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1016\/j.omtm.2017.11.007","article-title":"Emerging Issues in AAV-Mediated In Vivo Gene Therapy","volume":"8","author":"Colella","year":"2018","journal-title":"Mol Ther Methods Clin Dev"},{"issue":"23","key":"10.3233\/JND-210662_ref5","doi-asserted-by":"crossref","first-page":"12936","DOI":"10.1128\/JVI.01523-07","article-title":"The Mre11\/Rad50\/Nbs1 Complex Limits Adeno-Associated Virus Transduction and Replication","volume":"81","author":"Schwartz","year":"2007","journal-title":"J Virol"},{"key":"10.3233\/JND-210662_ref6","doi-asserted-by":"crossref","unstructured":"Dunbar CE , High KA , Joung JK , Kohn DB , Ozawa K , Sadelain M . Gene therapy comes of age. Science (80-.). 2018;359(6372).","DOI":"10.1126\/science.aan4672"},{"issue":"5","key":"10.3233\/JND-210662_ref7","doi-asserted-by":"crossref","first-page":"753","DOI":"10.1016\/j.hoc.2017.06.007","article-title":"Nonintegrating Gene T herapy Vectors","volume":"31","author":"Athanasopoulos","year":"2017","journal-title":"Hematol Clin NA"},{"issue":"3","key":"10.3233\/JND-210662_ref8","doi-asserted-by":"crossref","first-page":"723","DOI":"10.1016\/j.ymthe.2019.12.010","article-title":"AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer","volume":"28","author":"Verdera","year":"2020","journal-title":"Mol Ther"},{"key":"10.3233\/JND-210662_ref9","doi-asserted-by":"crossref","unstructured":"Little SR , et al. Poly-\u03b2 amino ester-containing microparticles enhance the activity of nonviral genetic vaccines. 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