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- 1From: Gene Therapy. (Vol. 18, Issue 7) Peer-ReviewedFragments of botulinum neurotoxin (BoNT) have been explored as potential targeting moieties and carriers of biomolecules into neurons, although with lower binding and translocation efficiency compared with intact...
- 2From: Gene Therapy. (Vol. 18, Issue 7) Peer-ReviewedCutaneous gene therapy can be envisioned through the use of keratinocyte stem cell clones in which retroviral genotoxic risks can be pre-assessed. While transactivation of cellular genes by the retroviral long terminal...
- 3From: Gene Therapy. (Vol. 18, Issue 7) Peer-ReviewedRecent success in clinical trials supports the use of adeno-associated viral (AAV) vectors for gene therapy of retinal diseases caused by defects in the retinal pigment epithelium (RPE). In contrast, evidence of the...
- 4From: Gene Therapy. (Vol. 18, Issue 7) Peer-ReviewedGene therapy has been applied to murine models of rheumatoid arthritis (RA) using a number of different strategies to downregulate inflammation in synovial joints. However, prolonged joint expression has been...
- 5From: Gene Therapy. (Vol. 18, Issue 7) Peer-ReviewedEven though it is known for more than one decade that antigen-encoding RNA can deliver antigenic information to induce antigen-specific immunity against cancer, the nature and mechanism of RNA uptake have remained...
- 6From: Gene Therapy. (Vol. 18, Issue 7) Peer-ReviewedWe previously have described a model of multiple sclerosis (MS) in which constitutive expression of murine interleukin (IL)-2 by herpes simplex virus type 1 (HSV-1) (HSV-IL-2) causes central nervous system (CNS)...
- 7From: Gene Therapy. (Vol. 18, Issue 7) Peer-ReviewedGene transfer to central nervous system (CNS) has been approached using various vectors. Recombinant SV40-derived vectors (rSV40s) transduce human neurons and microglia effectively in vitro and in rodent brains in vivo,...
- 8From: Gene Therapy. (Vol. 18, Issue 7) Peer-ReviewedExperimental autoimmune encephalomyelitis (EAE) is an autoimmune inflammation of the central nervous system and is used as the experimental model of multiple sclerosis (MS). The exact mechanism behind the disease is...
- 9From: Gene Therapy. (Vol. 18, Issue 7) Peer-ReviewedLegitimate uses of gene transfer technology can benefit from sensitive detection methods to determine vector biodistribution in pre-clinical studies and in human clinical trials, and similar methods can detect...
- 10From: Gene Therapy. (Vol. 18, Issue 7) Peer-ReviewedSmall interfering RNAs (siRNAs) are emerging as promising therapeutic tools. However, the widespread clinical application of such molecules as modulators of gene expression is still dependent on several aspects that...
- 11From: Gene Therapy. (Vol. 18, Issue 7) Peer-ReviewedMatrix metalloproteinases (MMPs) are a family of proteinases known to have a role in cell migration. In the present study, we evaluated the role of MMP-2 on tropism of human umbilical cord blood-derived stem cells...
- 12From: Gene Therapy. (Vol. 18, Issue 7) Peer-ReviewedTherapeutic RNA interference (RNAi) has emerged as a promising approach for the treatment of many incurable diseases, including cancer, infectious disease or neurodegenerative disorders. Demonstration of efficacy and...