The Pros And Cons Of Gene Therapy

Despite the best efforts of researchers on gene therapy, however, gene therapy has been related with numerous challenges over the last few decades. One challenge facing the field is the development of vector formulations to achieve optimal vector safety, stability and efficacy (Wright, Qu, Tang & Sommer, 2003). Li and Huang (2000) stated that successful gene therapy is largely dependent on the development of the vector that can efficiently deliver a gene to target cell with minimal toxicity. Nonviral and viral vectors are largely used in gene therapy. It is important to make the vector safe and efficient because delivering unsafe vector into a cell can cause severe health problems to patients. Other than that, gene therapy also can trigger

This challenge can be demonstrated by the story of Jesse Gelsinger, who had a rare liver disorder and participated in a 1999 gene therapy trial. Unfortunately, he died because of complications from an inflammatory response shortly after receiving a dose of experimental adenovirus vector. Another challenge in gene therapy is the ability to cause mutations. The new gene might be inserted into the wrong location in the DNA which will cause changes in the DNA. This has occurred in gene therapy trials that aimed at treating X-linked Severe Combined Immune Deficiency (SCID). Retrovirus was used as a corrective transgene to transduce the hematopoietic stem cells; however, it led to the development of T cell leukemia. It has been found that the newly transferred gene had stitched into a gene that normally helps regulate the rate at which cells divide and as a result, the cells began to divide out of control causing leukemia. Thus, developing a safer ways to introduce genes is crucial and some newer vectors have features that target DNA integration to specific safe places in the genome where it won’t cause