Adeno-associated virus vector as a platform for gene therapy delivery
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …
treatment of a variety of human diseases. Recent advances in developing clinically …
TLR9-activating CpG-B ODN but not TLR7 agonists triggers antibody formation to factor IX in muscle gene transfer
JSS Butterfield, M Biswas, JL Shirley… - Human gene therapy …, 2019 - liebertpub.com
Innate immune signals that promote B cell responses in gene transfer are generally ill-
defined. In this study, we evaluate the effect of activating endosomal Toll-like receptors 7, 8 …
defined. In this study, we evaluate the effect of activating endosomal Toll-like receptors 7, 8 …
A Genome-Wide Knock-Out Screen Identifies Novel Host Cell Entry Factor Requirements for Divergent Adeno-Associated Virus Serotypes
AM Dudek - 2019 - search.proquest.com
Abstract Adeno-Associated Virus (AAV) is a non-pathogenic virus that has been harnessed
as a vector system for therapeutic gene transfer. Despite decades of research on AAV as a …
as a vector system for therapeutic gene transfer. Despite decades of research on AAV as a …
Mécanismes moléculaires impliqués dans l'efficacité de transduction des vecteurs AAV dans le muscle dystrophique
J Chassagne - 2019 - theses.hal.science
La Dystrophie Musculaire de Duchenne (DMD) est une maladie génétique causée par
l'absence de dystrophine et provoquant une dégénérescence musculaire sévère. Aucun …
l'absence de dystrophine et provoquant une dégénérescence musculaire sévère. Aucun …
Pre-Clinical Evaluation of a Vaccination Strategy to Induce Liver Resident Memory T Cells against Hepatitis C Virus
ZA Mekonnen - 2019 - digital.library.adelaide.edu.au
Hepatitis C virus (HCV) is a significant contributor to the global burden of disease with at
least 71 million individuals persistently infected with the virus. Although effective anti-viral …
least 71 million individuals persistently infected with the virus. Although effective anti-viral …
CRISPR/Cas9 genome editing for the generation of YB1 knockout human and insect cell lines for the production of recombinant Adeno-associated virus vectors
K Miah - 2019 - centaur.reading.ac.uk
The use of Adeno-associated viral (AAV) vectors for gene therapy has been most promising
because of their safety profile; however, current production methods limit desirable amounts …
because of their safety profile; however, current production methods limit desirable amounts …