P
Pascal van der Wegen
Researcher at Erasmus University Rotterdam
Publications - 6
Citations - 268
Pascal van der Wegen is an academic researcher from Erasmus University Rotterdam. The author has contributed to research in topics: Small hairpin RNA & Genetic enhancement. The author has an hindex of 5, co-authored 6 publications receiving 254 citations.
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Journal ArticleDOI
Simultaneous Targeting of HCV Replication and Viral Binding with a Single Lentiviral Vector Containing Multiple RNA Interference Expression Cassettes
Scot D. Henry,Pascal van der Wegen,Herold J. Metselaar,Hugo W. Tilanus,Bob J. Scholte,Luc J. W. van der Laan +5 more
TL;DR: Lentiviral delivery of multiple shRNA, inhibiting HCV in a specific, IFN-independent, manner is demonstrated, suggesting the targeting of multiple viral and host cell elements simultaneously by RNAi could increase the potency of antiviral gene therapies.
Journal ArticleDOI
Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype.
Niek P. van Til,Merel Stok,Fatima S. F. Aerts Kaya,Monique C. de Waard,Elnaz Farahbakhshian,Trudi P. Visser,Marian A. Kroos,Edwin H. Jacobs,Monique Willart,Pascal van der Wegen,Bob J. Scholte,Bart N. Lambrecht,Dirk J. Duncker,Ans T. van der Ploeg,Arnold J. J. Reuser,Monique M A Verstegen,Gerard Wagemaker +16 more
TL;DR: It is concluded that ex vivo HSC gene therapy is a treatment option worthwhile to pursue on the basis of the prominent and sustained therapeutic efficacy without adverse events in mice.
Journal ArticleDOI
Successful treatment of UGT1A1 deficiency in a rat model of Crigler-Najjar disease by intravenous administration of a liver-specific lentiviral vector.
Pascal van der Wegen,Rogier Louwen,A. M. Ali Imam,Ruvalic M. Buijs-Offerman,Maarten Sinaasappel,Frank Grosveld,Bob J. Scholte +6 more
TL;DR: A self-inactivating lentiviral vector (LV-ALBUGT) is constructed that expresses the human bilirubin UDP-glucuronosyltransferase (UGT1A1) from a liver-specific promoter, involved in the clearance of heme metabolites in the liver.
Journal ArticleDOI
Lentiviral small hairpin RNA delivery reduces apical sodium channel activity in differentiated human airway epithelial cells
Jamil Aarbiou,Elena Copreni,Ruvalic M. Buijs-Offerman,Pascal van der Wegen,Stefano Castellani,Annalucia Carbone,Francesca Tilesi,Piera Assunta Fradiani,Pieter S. Hiemstra,Guelnihal Yueksekdag,Anna Diana,Joseph Rosenecker,Fiorentina Ascenzioni,Massimo Conese,Bob J. Scholte +14 more
TL;DR: Proof‐of-principle is shown for ENaC inhibition by lentiviral‐mediated RNA interference in mice model of cystic fibrosis.
Journal ArticleDOI
Hydroxyethyl starch-based preservation solutions enhance gene therapy vector delivery under hypothermic conditions
Scot D. Henry,Pascal van der Wegen,Herold J. Metselaar,Bob J. Scholte,Hugo W. Tilanus,Luc J. W. van der Laan +5 more
TL;DR: It is demonstrated that effective vector delivery can be achieved under conditions of hypothermic liver perfusion, and UW provides superior transduction to hepatocytes over nonstarch solutions.