RNA interference (RNAi) mediated by small interfering RNAs (siRNAs) enables knockdown of a gene of choice, executing the logical operation: silence gene Y. The fact that the siRNA is constitutively active is a significant limitation, making it difficult to confine knockdown to a specific locus and time. To achieve spatiotemporal control over silencing, we seek to engineer small conditional RNAs...

Gene transfer vectors based on adeno-associated virus (AAV) appear promising because of their high transduction frequencies regardless of cell cycle status and ability to integrate into chromosomal DNA. We tested AAV-mediated gene transfer into a panel of human bone marrow or umbilical cord-derived CD34+ hematopoietic progenitor cells, using vectors encoding several transgenes under the control...

The muscular dystrophies are a group of devastating genetic disorders that affect both skeletal and cardiac muscle. An effective gene therapy for these diseases requires bodywide muscle delivery. Tyrosine mutant adeno-associated virus (AAV) has been considered as a class of highly potent gene transfer vectors. Here, we tested the hypothesis that systemic delivery of tyrosine mutant AAV can resu...

Streptococcus bovis HJ50 produces a lacticin 481-like 33-amino-acid-residue lantibiotic, designated bovicin HJ50. bovK-bovR in the bovicin HJ50 biosynthetic gene cluster is predicted to be a two-component signal transduction system involved in sensing signals and regulating gene expression. Disruption of bovK or bovR resulted in the abrogation of bovicin HJ50 production, suggesting both genes p...

Gene transfer to photoreceptor cells may provide a means for arresting the retinal degeneration that is characteristic of many inherited causes of blindness, including retinitis pigmentosa (RP). However, transduction of photoreceptors has to date been inefficient, and further limited by toxicity and immune responses directed against vector-specific proteins. An alternative vector system based o...

In this study, in order to facilitate and accelerate the production of eukaryotic protein alpha 1-antitrypsin (AAT) with correct post-translational modifications, a protein production system based on the transduction of CHO and COS-7 cells using lentiviral vectors was developed. Human AAT cDNA was cloned into a replication-defective lentiviral vector. The transgene AAT-Jred chimer was transferr...

Many crucial components of signal transduction, developmental, and metabolic pathways have functionally redundant copies. Further, these redundancies show surprising evolutionary stability over prolonged time scales. We propose that redundancies are not just archeological leftovers of ancient gene duplications, but rather that synergy arising from feedback between redundant copies may serve as ...

in this study, in order to facilitate and accelerate the production of eukaryotic protein alpha 1-antitrypsin (aat) with correct post-translational modifications, a protein production system based on the transduction of cho and cos-7 cells using lentiviral vectors was developed. human aat cdna was cloned into a replication-defective lentiviral vector. the transgene aat-jred chimer was transferr...

The signal transduction processes involved in the regulation of SAMDC gene expression by blue and red light were examined using pharmacological inhibitors of signalling pathways. Calcium and calmodulin positively regulated SAMDC gene expression in red light, whereas in blue light they regulated negatively. These results indicate that calcium homeostasis is involved in both red and blue light in...