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- 1From: Gene Therapy. (Vol. 20, Issue 6) Peer-ReviewedThe development of effective strategies for gene therapy has been hampered by difficulties verifying transgene delivery in vivo and quantifying gene expression non-invasively. Magnetic resonance imaging (MRI) offers...
- 2From: Gene Therapy. (Vol. 20, Issue 6) Peer-ReviewedMicrotubule-based transport is required for plasmid translocation to the nucleus during transfections, and having stable structures could enhance this movement. In previous studies, in which the cytoskeleton was...
- 3From: Gene Therapy. (Vol. 20, Issue 6) Peer-ReviewedReplication-deficient retroviruses have been successfully utilized as vectors, offering an efficient, stable method of therapeutic gene delivery. Many examples exist proving this mode of integrative gene transfer is...
- 4From: Gene Therapy. (Vol. 20, Issue 6) Peer-ReviewedNon-viral vesicle composing of low-molecular weight polyethylenimine-conjugated stearic acid-g-chitosan oligosaccharide (CSOSA-g-PEI) was synthesized for gene delivery and therapy. The synthesized CSOSA-g-PEI had good...
- 5From: Gene Therapy. (Vol. 20, Issue 6) Peer-ReviewedAdoptive T-cell therapies have shown significant promise in the treatment of cancer and viral diseases. One approach, which introduces antigen-specific T-cell receptors (TCRs) into ex vivo activated T cells, is designed...
- 6From: Gene Therapy. (Vol. 20, Issue 6) Peer-ReviewedIn vivo delivery is a major barrier to the use of molecular tools for gene modification. Here we demonstrate site-specific gene editing of human cells in vivo in hematopoietic stem cell-engrafted NOD.Cg-[Prkdc.sup.scid]...
- 7From: Gene Therapy. (Vol. 20, Issue 6) Peer-ReviewedThe palindromic terminal repeats (TRs) of adeno-associated virus (AAV) form DNA hairpins (HPs) are essential for replication and for priming the conversion of single-stranded virion DNA to double strand. In recombinant...
- 8From: Gene Therapy. (Vol. 20, Issue 6) Peer-ReviewedGene transfer to spinal cord cells may be crucial for therapy in spinal muscular atrophy, amyotrophic lateral sclerosis and spinal cord injury. Lentiviral vectors are efficient for transduction of a variety of cells,...
- 9From: Gene Therapy. (Vol. 20, Issue 6) Peer-ReviewedIn this study, we determined the in vitro and in vivo efficacy of sodium iodide symporter (NIS) gene transfer and the therapeutic potential of oncolytic virotherapy combined with radioiodine therapy using a...
- 10From: Gene Therapy. (Vol. 20, Issue 6) Peer-ReviewedCurrent therapeutic strategies for Huntington's disease (HD) are focused on symptom management of disease progression. Transcriptional dysregulation is one of the major characteristics in HD. REST is a transcriptional...
- 11From: Gene Therapy. (Vol. 20, Issue 6) Peer-ReviewedWith the long-term goal of developing a gene-based treatment for osteoarthritis (OA), we performed studies to evaluate the equine joint as a model for adeno-associated virus (AAV)-mediated gene transfer to large,...
- 12From: Gene Therapy. (Vol. 20, Issue 6) Peer-ReviewedWe previously compared the expression of several human factor VIII (fVIII) transgene variants and demonstrated the superior expression properties of B domain-deleted porcine fVIII. Subsequently, a hybrid human/porcine...