Prophylactic prednisolone promotes AAV5 hepatocyte transduction through the novel mechanism of AAV5 co-receptor PDGFRα upregulation and innate immune suppression.


Journal

Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950

Informations de publication

Date de publication:
21 Dec 2023
Historique:
medline: 21 12 2023
pubmed: 21 12 2023
entrez: 21 12 2023
Statut: aheadofprint

Résumé

Adeno-associated virus (AAV) vectors are used to deliver therapeutic transgenes, but host immune responses may interfere with transduction and transgene expression. We evaluated prophylactic corticosteroid treatment on AAV5-mediated expression in liver tissue. Wild-type C57BL/6 mice received 6x10

Identifiants

pubmed: 38126359
doi: 10.1089/hum.2023.065
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Auteurs

Britta Handyside (B)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; britta.handyside@bmrn.com.

Lening Zhang (L)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; LZhang@bmrn.com.

Bridget Yates (B)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; BYates@bmrn.com.

Lin Xie (L)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; lin.xie@bmrn.com.

Mohamed Ismail Ashrafali (MI)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; MohamedIsmail.Ashrafali@bmrn.com.

Ryan Murphy (R)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; ryan.murphy@bmrn.com.

Brian Baridon (B)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; bbaridon@bmrn.com.

Cheng Su (C)

BioMarin Pharmaceutical Inc, 10926, Global Clinical Sciences, Novato, California, United States; cheng.su@bmrn.com.

Taren Bouwman (T)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; Taren.Bouwman@bmrn.com.

Linley Mangini (L)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; linley.mangini@bmrn.com.

Jorden Tahquechi (J)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; Jorden.Tahqechi@bmrn.com.

Sandra Salcido (S)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; Sandra.Salcido@bmrn.com.

Wesley C Minto (WC)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; Wesley.minto@gmail.com.

William T Keenan (WT)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; Keenanw27@gmail.com.

Ioanna Ntai (I)

BioMarin Pharmaceutical Inc, 10926, Translational Sciences, Novato, California, United States; Ioanna.Ntai@bmrn.com.

Choong-Ryoul Sihn (CR)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; choongryoul.sihn@bmrn.com.

Sherry Bullens (S)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; SBullens@bmrn.com.

Stuart Bunting (S)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; sbunting@bmrn.com.

Sylvia Fong (S)

BioMarin Pharmaceutical Inc, 10926, Biology Research, Novato, California, United States; SFong@bmrn.com.

Classifications MeSH