{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,27]],"date-time":"2026-07-27T13:37:43Z","timestamp":1785159463898,"version":"3.55.0"},"reference-count":0,"publisher":"Scientific Archives","issue":"2","license":[{"start":{"date-parts":[[2023,8,28]],"date-time":"2023-08-28T00:00:00Z","timestamp":1693180800000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/www.scientificarchives.com\/about-us"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["RO1 NS095563"],"award-info":[{"award-number":["RO1 NS095563"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J Nanotechnol Nanomaterials"],"abstract":"<jats:p>A major obstacle to fulfilling the therapeutic promise of gene therapies for hereditary brain diseases, such as Huntington\u2019 Disease (HD), is the requirement for viral vectors and\/or an invasive delivery system (stereotaxic injection into brain or infusion into the intrathecal space). HD is an autosomal dominant neurodegenerative disease for which several clinical trials have demonstrated gene-lowering effects following intrathecal administration. These technical limitations have given impetus to the development of alternative non-invasive delivery systems for gene therapy of brain diseases. The overall objective of this review is to discuss the key features in the design of nanocarriers for intranasal administration of gene-therapy for HD, focusing primarily on our series of published work on the use of nanocarriers for gene therapy. Design and development of nanocarriers packaged with gene-lowering agents represents a significant advance towards non-invasive nose-to-brain delivery of gene therapy for HD and other hereditary brain disorders.<\/jats:p>","DOI":"10.33696\/nanotechnol.4.043","type":"journal-article","created":{"date-parts":[[2023,8,28]],"date-time":"2023-08-28T11:21:56Z","timestamp":1693221716000},"page":"55-69","source":"Crossref","is-referenced-by-count":8,"title":["Key Features in the Design and Function of Nanocarriers for Intranasal Administration of Gene Therapy in Huntington Disease"],"prefix":"10.33696","volume":"4","author":[{"given":"Oksana","family":"Fihurka","sequence":"first","affiliation":[{"name":"Department of Neurology, University of South Florida, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephen","family":"Aradi","sequence":"additional","affiliation":[{"name":"Department of Neurology, University of South Florida, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vasyl","family":"Sava","sequence":"additional","affiliation":[{"name":"Department of Neurology, University of South Florida, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juan","family":"Sanchez-Ramos","sequence":"additional","affiliation":[{"name":"Department of Neurology, University of South Florida, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"18993","published-online":{"date-parts":[[2023,8,28]]},"container-title":["Journal of Nanotechnology and Nanomaterials"],"original-title":[],"language":"en","deposited":{"date-parts":[[2023,8,28]],"date-time":"2023-08-28T11:21:58Z","timestamp":1693221718000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.scientificarchives.com\/article\/key-features-in-the-design-and-function-of-nanocarriers-for-intranasal-administration-of-gene-therapy-in-huntington-disease"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,8,28]]},"references-count":0,"journal-issue":{"issue":"2","published-online":{"date-parts":[[2023,8,28]]}},"URL":"https:\/\/doi.org\/10.33696\/nanotechnol.4.043","relation":{},"ISSN":["2692-630X"],"issn-type":[{"value":"2692-630X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,8,28]]}}}