Current Gene Therapy - Volume 1, Issue 2, 2001

Despite the fact that adeno-associated virus type 2 (AAV2) is an extremely attractive gene therapy vector, its application has been limited to certain tissues such as muscle and the brain. In an attempt to broaden the array of target organs for this vector, molecular studies on the mechanism(s) of AAV transduction have expanded over the past several years. These studies have led to the development of innovative strategies capabl Read More

Human gene therapy promises to change the practice of medicine by treating the causes of disease rather than the symptoms. Since the first clinical trial made its debut ten years ago, there are over 400 approved protocols in the United States alone, most of which have failed to show convincing data of clinical efficacy. This setback is largely due to the lack of efficient and adequate gene transfer vehicles. With the rece Read More

Clinical gene therapy needs non invasive tools to evaluate the efficiency of gene transfer. This includes the evaluation of infection efficiency as well as the verification of successfull gene transfer in terms of gene transcription. These informations can be used for therapy planning, follow up studies in treated tumors and as an indicator of prognosis. Therapy planning is performed by the assessment of gene expression for exa Read More

In the last few years, significant advances in gene therapy have been made as a result of advances in many areas of molecular and cell biology, including the improvement of both viral and nonviral gene delivery systems, discovery of new therapeutic genes, better understanding of mechanism of disease progression, exploration of tissue specific promoter, receptor- and antibody-mediated targeting delivery, and dev Read More

The last 10 years have seen substantial progress in the development and application of nonviral vectors in gene therapy. Several novel nonviral methods have been developed that approach viruses with respect to transfection efficiency. A variety of nonviral delivery systems that can be used for gene therapy in different clinical settings are also available. In this review article, we will detail all of the major nonviral vectors that a Read More

Over the past decade, bone marrow transplantation has come to be considered an ideal therapeutic strategy for the treatment of certain diseases affecting the hematopoietic system such as hemophilia, and several clinical trials have Been performed. Although traditionally used for the treatment of lethal diseases, it is speculated that this approach could also be used in the treatment of non-lethal but much more comm Read More