The Target's the Thing

In the 1970s, many conceived of the Nixon administration's “War on Cancer” as the search for a single breakthrough that would allow for effective treatment of all forms of cancer, as if cancer were a single disease. Over the ensuing decades, effective prevention and/or therapies were devised for childhood acute leukemia, lymphomas, cervical carcinoma, breast cancer, colon cancer, and other malignancies. These advances were grounded in a fundamental understanding that just as the biology of each of these organ systems was unique, so too was the biology of each cancer.

In a similar fashion, the dramatic increase in the success of clinical gene therapy over the past 8 to 10 years has been due not to a single technological advance but to a series of individual advances for safe and effective gene transfer to each organ system. Human Gene Therapy is pleased to initiate a series of review articles devoted to gene transfer to each of a series of tissues, organs, and organ systems that serve as targets for therapeutic gene transfer.

In this issue, we kick off this series with a review article from Dr. Nathalie Cartier et al. on advances in gene therapy for the central nervous system (CNS). The CNS is, in many ways, the holy grail of gene therapy, because the brain is the home of our consciousness and is considered by many as the seat of the soul. The imperative of developing effective gene therapy for diseases of the CNS is of ever-increasing priority as the aging demographic of baby-boomers promises to deliver an increasing burden of neurodegenerative diseases to the healthcare systems of the United States and Europe. The editorial team at Human Gene Therapy sincerely hopes that our readership will benefit from these contributions and similar reviews scheduled to appear in subsequent issues throughout the remainder of the year.