Lentiviral vectors are widely used in the field of gene therapy as an effective method for permanent gene delivery. While current methods of producing small scale vector batches for research purposes depend largely on culture flasks, the emergence and popularity of lentiviral vectors in translational, preclinical and clinical research has demanded their production on a much larger scale, a task...

Addressing the envelope for gene delivery ---------------------------------------------------------------------------------------------------------------Richard J. O’Reilly1 1MEMORIAL SLOAN-KETTERING CANCER CENTER In this issue of Blood, Zhou et al describe a strategy for modifying the envelope proteins of a lentiviral vector to display a single chain antibody fragment, or ScFv, specific for CD...

Efficient gene delivery in the central nervous system (CNS) is important in studying gene functions, modeling neurological diseases and developing therapeutic approaches. Lentiviral vectors are attractive tools in transduction of neurons and other cell types in CNS as they transduce both dividing and non-dividing cells, support sustained expression of transgenes, and have relatively large packa...

Hematopoietic stem cell gene therapy requires the use of integrating retroviral vectors in order to stably transmit a therapeutic gene to mature blood cells. Human clinical trials have shown that some vector integration events lead to disrupted regulation of proto-oncogenes resulting in disordered hematopoiesis including T-cell leukemia. Newer vectors have been designed to decrease the incidenc...

C-C chemokine receptor type 5 (CCR5) is utilized by human immunodeficiency virus (HIV) as a co-receptor for cell entry. Suppression of the CCR5 gene by artificial microRNAs (amiRNAs) could confer cell resistance. In previous work, we created a lentivector that encoded the polycistron of two identical amiRNAs that could effectively suppress CCR5. However, tandem repeats in lentiviral vectors led...

The delivery of adipose-derived stem cells (ADSCs) for promoting tissue repair has become a potential new therapy, while hepatocyte growth factor (HGF) is an important growth factor with angiogenic, anti-fibrotic, and anti-inflammatory benefits. In this paper, hADSCs were separated, cultured and identified based on the expression of cell surface antigens and multiple differentiation potential. ...

Mucopolysaccharidosis type I (MPS I) is a lysosomal disease caused by α-Liduronidase (IDUA) deficiency and subsequent accumulation of glycosaminoglycan (GAG) heparan sulfate and dermatan sulfate. Lentiviral vector encoding correct IDUA sequence could be used for gene therapy treating MPS I disease. Previous study in our lab showed that intravenous injection of a lentiviral vector (CSP1) into ne...

BACKGROUND/AIMS Stem cell transplantation and gene therapies have been shown to attenuate myocardial dysfunction after myocardial infarction (AMI) in different acute and chronic animal models. The aim of this study was to assess the potential therapeutic efficacy of endothelial NO synthases (eNOS)-expressing endothelial progenitor cells (EPCs) on infarcted hearts. METHODS Lentiviral eNOS-infe...

BACKGROUND During the RNA encapsidation process of human immunodeficiency virus (HIV) viral genomic, unspliced RNA (gRNA) is preferentially incorporated into assembling virions. However, a certain amount of spliced viral transcripts can also be detected in viral particles. Recently, we observed that nuclear export of HIV and lentiviral vector gRNA by Rev is required for efficient encapsidation....