First pilot study of intravenous rAAV-PAX6 gene therapy increases retinal-ganglion-cell-layer thickness and Notch1 transcription in a mouse model of aniridia.
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| Title: | First pilot study of intravenous rAAV-PAX6 gene therapy increases retinal-ganglion-cell-layer thickness and Notch1 transcription in a mouse model of aniridia. |
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| Authors: | Djaksigulova D; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada., Kaad SG; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada., Korecki AJ; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada., Lam SL; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada., Lengyell TC; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada., Simpson EM; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada. simpson@cmmt.ubc.ca.; Department of Medical Genetics, The University of British Columbia, Vancouver, BC, Canada. simpson@cmmt.ubc.ca. |
| Source: | Gene therapy [Gene Ther] 2026 Mar 09. Date of Electronic Publication: 2026 Mar 09. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 41796252 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: First pilot study of intravenous rAAV-PAX6 gene therapy increases retinal-ganglion-cell-layer thickness and Notch1 transcription in a mouse model of aniridia. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Djaksigulova+D%22">Djaksigulova D</searchLink>; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada.<br /><searchLink fieldCode="AU" term="%22Kaad+SG%22">Kaad SG</searchLink>; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada.<br /><searchLink fieldCode="AU" term="%22Korecki+AJ%22">Korecki AJ</searchLink>; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada.<br /><searchLink fieldCode="AU" term="%22Lam+SL%22">Lam SL</searchLink>; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada.<br /><searchLink fieldCode="AU" term="%22Lengyell+TC%22">Lengyell TC</searchLink>; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada.<br /><searchLink fieldCode="AU" term="%22Simpson+EM%22">Simpson EM</searchLink>; Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, BC, Canada. simpson@cmmt.ubc.ca.; Department of Medical Genetics, The University of British Columbia, Vancouver, BC, Canada. simpson@cmmt.ubc.ca. – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%229421525%22">Gene therapy</searchLink> [Gene Ther] 2026 Mar 09. <i>Date of Electronic Publication: </i>2026 Mar 09. – Name: TypePub Label: Publication Type Group: TypPub Data: Journal Article – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Nature+Publishing+Group%22">Nature Publishing Group </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>9421525 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1476-5462 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2209697128%22">09697128 </searchLink><i>NLM ISO Abbreviation: </i>Gene Ther <i>Subsets: </i>MEDLINE |
| PLink | https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=41796252 |
| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1038/s41434-026-00605-5 Languages: – Code: eng Text: English Titles: – TitleFull: First pilot study of intravenous rAAV-PAX6 gene therapy increases retinal-ganglion-cell-layer thickness and Notch1 transcription in a mouse model of aniridia. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Djaksigulova D – PersonEntity: Name: NameFull: Kaad SG – PersonEntity: Name: NameFull: Korecki AJ – PersonEntity: Name: NameFull: Lam SL – PersonEntity: Name: NameFull: Lengyell TC – PersonEntity: Name: NameFull: Simpson EM IsPartOfRelationships: – BibEntity: Dates: – D: 09 M: 03 Text: 2026 Mar 09 Type: published Y: 2026 Identifiers: – Type: issn-electronic Value: 1476-5462 Titles: – TitleFull: Gene therapy Type: main |
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