This year could mark the point at which gene therapy moves from the lab into commercialisation, the editor-in-chief of Human Gene Therapy has predicted.
In a new commentary piece, James Wilson, MD, PhD, suggested that gene therapies have proved their effectiveness and feasibility, and must be made available to patients in need.
He writes: "2012 will usher in an era of commercial development of gene therapy that, although likely to begin slowly, will quickly gather momentum."
Dr Wilson drew attention to a recent report on gene therapy proving effective in treating haemophilia B and argues it could form a realistic alternative to the regular, lifelong protein replacement infusions patients currently have to use.
Work is also being undertaken on a gene therapy treatment for haemophilia A, although both are currently in the early stages of clinical testing.
Recent advancements ha also sought to overcome the toxicity and efficiency of gene therapy delivery, with scientists discovering short spiral-shaped proteins can efficiently deliver DNA segments to cells.
Published in the journal Angewandte Chemie, the paper details the creation of a helical polypeptide that can outperform many commercial agents in the delivery of DNA to cells that are even the most difficult to transfect.
Professor Jianjun Cheng from the University of Illinois explained: "The polypeptide that we designed, synthesized and used in this study has very high efficiency and also well-controlled toxicities," and is expected to prove useful in the further development of gene therapies.
Terence R. Flotte, MD, from the Departments of Pediatrics and Microbiology & Physiologic Systems, University of Massachusetts Medical School, said the development of gene therapy would mark a return on the investment in biomedical research and genetic sequencing.
"Dr. Wilson's comments strike at the heart of fulfilling that promise. Now that the technology is working, the next phase is to develop commercially viable models for gene therapy in the health care marketplace, so that these therapies can be delivered to the patients who need them," he added.



Be part of the Fisher Scientific social scene