Current Gene Therapy - Volume 3, Issue 5, 2003

Recently, stem cell research has attracted considerable attention because it could be used for the regeneration of damaged organs that are untreatable by conventional techniques, and several stem cells (or progenitor cells), such as endothelial stem cells and neural stem cells have been discovered. Following the progression of this field of research, the potential for stem cell gene therapy has increased and several therap Read More

Genomics and proteomics have unveiled a plethora of protein-protein interactions that may control cellular processes involved in disease development. Many of these interactions involve non-traditional candidate targets (i.e., neither enzymes nor cell surface receptors / channels). To date, non-traditional targets have largely been ignored by the pharmaceutical industry or have failed to lead to drugs. This review focus Read More

A conceptual breakthrough in gene therapy would be gene transfer vector that could be systemically applied, allowing targeted gene transfer into a predetermined cell type. The host range of a retroviral vector is determined by the interaction of the viral envelope glycoprotein (Env) and the retrovirus receptor on the surface of the host cell. In this review, we describe the current efforts to engineer targeted envelope glycopro Read More

The use of anti-gene agents to disrupt the expression of disease-related genes could potentially be of utility in the treatment of a large number of illnesses, including most neoplasms. Traditional anti-gene agents include antisense oligonucleotides and ribozymes. Recent observations have provided evidence for another promising anti-gene technology-RNA interference (RNAi), in which the introduced double-stranded RNA (d Read More

Cardiovascular disease remains a major cause of morbidity and mortality in modern societies. While contemporary treatment modalities are making steady inroads to reduce this disease burden there remains a pressing need to vigorously explore novel therapeutic strategies. Rapid advances in our understanding of molecular pathology and the evolution of increasingly efficient gene transfer technology offer the imm Read More

Gene therapy is a translational science, with the ultimate goal of cancer gene therapy research being to develop effective and safe treatments for patients. In the new millennium, it is imperative to tailor a therapeutic strategy for a particular disease, based on clinical management issues. The desirable regulatory features and therapeutic strategies need to be fully considered before proceeding with molecular engineering of th Read More

Although specific delivery to tissues and unique cell types in vivo has been demonstrated for many non-viral vectors, current methods are still inadequate for human applications, mainly because of limitations on their efficiencies. All the steps required for an efficient receptor-mediated gene transfer process may in principle be exploited to enhance targeted gene delivery. These steps are: DNA / vector binding, internaliz Read More

Protein transduction domains (PTDs, sometimes termed cell permeable proteins (CPP) or membrane translocating sequences (MTS)) are small peptides that are able to ferry much larger molecules into cells independent of classical endocytosis. This property makes PTDs ideal tools to transfer proteins and other molecules into living cells for research purposes. The mechanism by which this internalization takes place is Read More