Show simple item record

dc.contributor.authorGruntman, Alisha
dc.contributor.authorSu, Lin
dc.contributor.authorFlotte, Terence R.
dc.date2022-08-11T08:10:12.000
dc.date.accessioned2022-08-23T16:59:22Z
dc.date.available2022-08-23T16:59:22Z
dc.date.issued2017-03-01
dc.date.submitted2017-06-01
dc.identifier.citationHum Gene Ther. 2017 Mar;28(3):228-230. doi: 10.1089/hum.2017.037. <a href="https://doi.org/10.1089/hum.2017.037">Link to article on publisher's site</a>
dc.identifier.issn1043-0342 (Linking)
dc.identifier.doi10.1089/hum.2017.037
dc.identifier.pmid28319444
dc.identifier.urihttp://hdl.handle.net/20.500.14038/43567
dc.description.abstractIn order to pursue a clinical gene therapy for a human neurologic disease, it is often necessary to perform proof-of-concept trials in mouse models of that disease. In order to demonstrate a potential clinical efficacy, one must be able to select an appropriate vector and route of delivery for the appropriate age group in the disease model. Since many diseases require correction early in life, investigators often need to deliver recombinant adeno-associated viral (rAAV) vectors to neonatal mice. Herein, general central nervous system expression patterns of nuclear GFP following delivery of rAAV by three different routes are reported.
dc.language.isoen_US
dc.relation<a href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&list_uids=28319444&dopt=Abstract">Link to Article in PubMed</a>
dc.relation.urlhttps://doi.org/10.1089/hum.2017.037
dc.subjectGenetics and Genomics
dc.subjectNervous System Diseases
dc.subjectPediatrics
dc.subjectTherapeutics
dc.titleRetro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups
dc.typeJournal Article
dc.source.journaltitleHuman gene therapy
dc.source.volume28
dc.source.issue3
dc.identifier.legacycoverpagehttps://escholarship.umassmed.edu/peds_pp/138
dc.identifier.contextkey10236425
html.description.abstract<p>In order to pursue a clinical gene therapy for a human neurologic disease, it is often necessary to perform proof-of-concept trials in mouse models of that disease. In order to demonstrate a potential clinical efficacy, one must be able to select an appropriate vector and route of delivery for the appropriate age group in the disease model. Since many diseases require correction early in life, investigators often need to deliver recombinant adeno-associated viral (rAAV) vectors to neonatal mice. Herein, general central nervous system expression patterns of nuclear GFP following delivery of rAAV by three different routes are reported.</p>
dc.identifier.submissionpathpeds_pp/138
dc.contributor.departmentHorae Gene Therapy Center
dc.contributor.departmentDepartment of Pediatrics
dc.source.pages228-230


This item appears in the following Collection(s)

Show simple item record