The Technology of Gene Therapy

T wenty five years of gene therapy research is paying off! We are seeing an upswing in clinical trials using second generation vector technologies with some evidence of success. It is a great time to be active in gene therapy. To this end, the leadership of Human Gene Therapy and its affiliated societies continually reassesses how we can best contribute to the evolution of the field. In contrast to most medical-based journals/societies, which are organized around specific types of diseases, the common thread for gene therapy is science and technology. Human Gene Therapy welcomes a broad scope of topics in our research articles/brief reports/reviews/commentaries/editorials. Beginning with this issue, we plan to place a special emphasis on the technology of gene therapy in a section called “Methods.”

The progress we have seen in gene therapy is encouraging although we still have a ways to go before this therapeutic approach becomes incorporated into the practice of medicine. Increased activity in translation is clearly warranted although more basic science and better technology are needed. For example, one of the most commonly cited concerns regarding the successful commercialization of gene therapy relates to the feasibility and cost of large-scale vector production.

We encourage submission of manuscripts to the Journal that describe key technological advances. These manuscripts can cover a wide range of topics from techniques for detecting transgene expression, to improved vectors to better manufacturing methods and product testing assays. The format for a Methods article will be the same as for a regular manuscript, although it will carry the “Methods” designation. Reviews, commentaries and editorials related to the technology of gene therapy are also encouraged.

Please do not underestimate the potential interest in an important methods article. Some of the most highly cited articles that have been published in the Journal have described key technological advances. We look forward to receiving your methods manuscripts and will make sure that those with the potential for a high impact receive an expedited review.