Tag: <span>gene transfer</span>

Current in vivo gene therapy (GT) approaches are beginning to demonstrate significant clinical and safety limitations that may ultimately reduce their therapeutic utility. In particular, the potential for systemic toxicity due to the antigenicity of the gene transfer vector, the prospect of insertional mutagenesis/oncogenesis during gene transfer, and the possibility of germ line transfer of the transgene are issues raising concern. One promising alternative to gene therapy that mitigates these clinical and safety issues is gene-based cell therapy (GBCT), in which autologous cells are removed from a patient and modified ex vivo for a desired characteristic prior to reimplantation. By transferring the transgene ex vivo, many of the issues surrounding the in vivo use of the transfer vectors are reduced and issues surrounding germ line transfer can be practically eliminated…

Cell & Gene Therapy

The modern era of interest in gene transfer as a methodology for treating disease began around 1985 with the first use and publication of mouse-based retroviruses that could transduce human cells. In fact, the use of gene transfer as a clinically useful method is probably older than any other therapy commonly used today — it forms the basis for the vaccinia vaccination against smallpox, popularized in Western medicine by Jenner. Another antecedent is phage therapy for bacterial infection, which was largely but not completely superseded by antibiotics (although it may make a comeback in this era of drug-resistant pathogenic bacterial strains.) Other examples include the other live viral vaccines: measles/mumps/rubella, polio, varicella, tuberculosis, influenza, the use of bacillus Calmette-Guérin (BCG) as a therapeutic for bladder cancer bone marrow transplants, and even the use of maggots to clean wounds…

Cell & Gene Therapy Viral Vectors