Efficient recombinase-mediated cassette exchange in hPSCs to study the hepatocyte lineage reveals AAVS1 locus-mediated transgene inhibition

L Ordovás, R Boon, M Pistoni, Y Chen, E Wolfs, W Guo… - Stem cell reports, 2015 - cell.com
Tools for rapid and efficient transgenesis in" safe harbor" loci in an isogenic context remain
important to exploit the possibilities of human pluripotent stem cells (hPSCs). We created …

Sustained expression from DNA vectors

SP Wong, O Argyros, RP Harbottle - Advances in genetics, 2015 - Elsevier
DNA vectors have the potential to become powerful medical tools for treatment of human
disease. The human body has, however, developed a range of defensive strategies to detect …

MicroRNA regulation of oncolytic herpes simplex virus-1 for selective killing of prostate cancer cells

CYF Lee, PS Rennie, WWG Jia - Clinical Cancer Research, 2009 - AACR
Purpose: Advanced castration-resistant prostate cancer, for which there are few treatment
options, remains one of the leading causes of cancer death. MicroRNAs (miRNA) have …

HSV as a vector in vaccine development and gene therapy

P Marconi, R Argnani, AL Epstein… - Pharmaceutical …, 2009 - Springer
The verydeepknowledge acquiredon the genetics and molecularbiologyof herpessimplex
virus (HSV), major human pathogenwhoselifestyle is basedon a long-termdual interaction …

Optimised coding sequence and promoter

A Nathwani, N Ward, A Thrasher, E Tuddenham… - US Patent …, 2016 - Google Patents
An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter
may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood …

Advances in liver-directed gene therapy for hepatocellular carcinoma by non-viral delivery systems

B Ding, T Li, J Zhang, L Zhao, G Zhai - Current gene therapy, 2012 - ingentaconnect.com
Hepatocellular carcinoma (HCC) is a malignancy with a high mortality. Gene therapy
provides a promising way for the treatment of HCC. Efficient gene delivery system, suitable …

HSV as a vector in vaccine development and gene therapy

P Marconi, R Argnani, AL Epstein, R Manservigi - Human Vaccines, 2008 - Taylor & Francis
The very deep knowledge acquired on the genetics and molecular biology of herpes
simplex virus (HSV), major human pathogen whose lifestyle is based on a long-term dual …

Progress and prospects: biological properties and technological advances of herpes simplex virus type 1-based amplicon vectors

AL Epstein - Gene therapy, 2009 - nature.com
The last two years have seen significant advances in our understanding of the cellular
innate responses elicited or activated by the entry of amplicon particles, which may, in part …

Novel tumour‐specific promoters for transcriptional targeting of hepatocellular carcinoma by herpes simplex virus vectors

P Foka, A Pourchet… - The journal of gene …, 2010 - Wiley Online Library
Background Hepatocellular carcinoma (HCC) is a cancer of poor prognosis, with limited
success in patient treatment, which it makes an excellent target for gene therapy and viral …

Human hepatocellular carcinoma: expression profiles-based molecular interpretations and clinical applications

KM Hui - Cancer letters, 2009 - Elsevier
Primary liver cancer is the fifth most common cancer worldwide and hepatocellular
carcinoma (HCC) accounts for over 85% of all primary liver cancers. The clinical …