Current Gene Therapy - Volume 10, Issue 2, 2010

The last few years have seen the transfer of two decades of research into Chimeric Antigen Receptors (CARs) into clinical trials. Despite this extensive research, there is still a great deal of debate into the optimal design strategy for these, primarily, anti-cancer entities. The archetypal CAR consists of a single-chain antibody fragment, specific to a tumour- associated antigen, fused to a component of the T-cell receptor Read More

Gene therapy is a rapidly developing field in which recombinant nucleic acid sequences are introduced to individuals to regulate, repair, replace, add or delete a genetic sequence. Recombinant adeno-associated viral (AAV) vectors, especially AAV2, are frequently used in gene therapy. Knowledge on the biodistribution and potential shedding of AAV2 is crucial to evaluate the risks of infection with the viral vector for the p Read More

Gene therapy is a rapidly developing field in which recombinant nucleic acid sequences are introduced to individuals. Its therapeutic, prophylactic or diagnostic effect relates directly to the sequence it contains or to the product of genetic expression of this sequence. Recombinant adenoviral vectors (in particular HAdV-5 vectors) are frequently used in gene therapy. Knowledge on biodistribution and shedding is crucial in the ris Read More

Muscle electrotransfer covers the delivery of molecules to muscle tissue by means of electric pulses. This method has proven highly efficient in transferring, in particular, plasmid DNA to muscles, resulting in long-term expression of the transferred genes. DNA electrotransfer to muscle tissue has clinical potential within DNA vaccination, systemic delivery of therapeutic proteins and correction of gene defects in muscles. In the Read More

Small interfering RNAs (siRNA) are one of the most recent additions used to silence gene expression. At present, siRNA is the most extensively used gene-silencing technique over other nucleic-acid based approaches to treat diseases including cancer, hepatitis, respiratory disease, cardiovascular diseases, neuronal disease and autoimmune disease. However, systemic delivery of siRNA remains to be the biggest challenge to be ov Read More

Acute liver failure (ALF) is a life-threatening medical emergency and occurs when the liver rapidly loses its function within a short period. ALF can develop secondary to a variety of causes. Currently, the orthotopic liver transplantation is the “Gold Standard” therapy for the disease. However, due to the limited availability of donor organs and rapid progression of the disease, the mortality of ALF remains high. Therefore, it is im Read More