Immune responses to viral gene therapy vectors

JL Shirley, YP de Jong, C Terhorst, RW Herzog - Molecular Therapy, 2020 - cell.com
Several viral vector-based gene therapy drugs have now received marketing approval. A
much larger number of additional viral vectors are in various stages of clinical trials for the …

Lentiviral vector bioprocessing

C Perry, ACME Rayat - Viruses, 2021 - mdpi.com
Lentiviral vectors (LVs) are potent tools for the delivery of genes of interest into mammalian
cells and are now commonly utilised within the growing field of cell and gene therapy for the …

CRISPR/Cas9: principle, applications, and delivery through extracellular vesicles

K Horodecka, M Düchler - International Journal of Molecular Sciences, 2021 - mdpi.com
The establishment of CRISPR/Cas9 (clustered regularly interspaced short palindromic
repeats/CRISPR-associated protein 9) technology for eukaryotic gene editing opened up …

Progress and prospects: immune responses to viral vectors

S Nayak, RW Herzog - Gene therapy, 2010 - nature.com
Viral vectors are potent gene delivery platforms used for the treatment of genetic and
acquired diseases. However, just as viruses have evolved to infect cells efficiently, the …

Lentiviral vectors in gene therapy: their current status and future potential

D Escors, K Breckpot - Archivum immunologiae et therapiae …, 2010 - Springer
The concept of gene therapy originated in the mid twentieth century and was perceived as a
revolutionary technology with the promise to cure almost any disease of which the molecular …

Recent advances in lentiviral vector development and applications

J Mátrai, MKL Chuah, T VandenDriessche - Molecular therapy, 2010 - cell.com
Lentiviral vectors (LVs) have emerged as potent and versatile vectors for ex vivo or in vivo
gene transfer into dividing and nondividing cells. Robust phenotypic correction of diseases …

The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice

J Zhu, X Huang, Y Yang - The Journal of clinical …, 2009 - Am Soc Clin Investig
Recombinant adeno-associated viruses (AAVs) have been used widely for in vivo gene
therapy. However, adaptive immune responses to AAV have posed a significant hurdle in …

The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9–dependent innate immune responses in the liver

AT Martino, M Suzuki, DM Markusic… - Blood, The Journal …, 2011 - ashpublications.org
Although adeno-associated viral (AAV) vectors have been successfully used in hepatic gene
transfer for treatment of hemophilia and other diseases in animals, adaptive immune …

Co-delivery of nucleoside-modified mRNA and TLR agonists for cancer immunotherapy: Restoring the immunogenicity of immunosilent mRNA

R Verbeke, I Lentacker, L Wayteck, K Breckpot… - Journal of Controlled …, 2017 - Elsevier
This study reports on the design of mRNA and adjuvant-loaded lipid nanoparticles for
therapeutic cancer vaccination. The use of nucleoside-modified mRNA has previously been …

Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo

D Cesana, M Ranzani, M Volpin, C Bartholomae… - Molecular therapy, 2014 - cell.com
Self-inactivating (SIN) lentiviral vectors (LV) have an excellent therapeutic potential as
demonstrated in preclinical studies and clinical trials. However, weaker mechanisms of …