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BACKGROUND: Recent advances in functional genomics, gene therapy, tissue engineering, drug discovery and biopharmaceuticals production have been fostered by precise small-molecule-mediated fine-tuning of desired transgenes. METHODS: Capitalizing on well-ev ...
Gene therapy is an attractive approach for the treatment of a wide spectrum of liver diseases. Lentiviral vectors allow the stable integration of transgenes into the genome of nondividing differentiated cells including hepatocytes and could provide long-la ...
The secreted signaling molecule fibroblast growth factor 8 (Fgf8) is an essential component of certain embryonic signaling centers including the mid-hindbrain (isthmic) organizer, the first branchial arch (BA1), and the apical ectodermal ridge (AER). In th ...
We have generated a novel transgenic mouse model on a C57BL/ 6J genetic background that coexpresses KM670/ 671NL mutated amyloid precursor protein and L166P mutated presenilin 1 under the control of a neuron- specific Thy1 promoter element ( APPPS1 mice). ...
Analysis of genetically engineered mice is crucial for our understanding of the in vivo function of genes and proteins in the whole organism. This includes inactivation of a gene or the generation of specific mutations. The development of knockout and tran ...
First described about a decade ago, lentiviral vectors ('lentivectors') have emerged as potent and versatile tools of gene transfer for basic and applied research and offer exciting perspectives for the field of gene therapy. In the clinic, HIV-based vecto ...
Gene therapy by use of integrating vectors carrying therapeutic transgene sequences offers the potential for a permanent cure of genetic diseases by stable vector insertion into the patients' chromosomes. However, three cases of T cell lymphoproliferative ...
Recent advances in genomics, proteomics, and structural biology raised the general need for significant amounts of pure recombinant protein (r-protein). Because of the difficulty in obtaining in some cases proper protein folding in bacteria, several method ...
The Gam1 protein of the avian CELO adenovirus activates transcription through inhibition of histone deacetylase 1 (HDAC1). We investigated the effect of Gam1 on both transient and stable transgene expression in Chinese hamster ovary (CHO) cells, one of the ...
Mutations in Cu/Zn superoxide dismutase (encoded by SOD1), one of the causes of familial amyotrophic lateral sclerosis (ALS), lead to progressive death of motoneurons through a gain-of-function mechanism. RNA interference (RNAi) mediated by viral vectors a ...