Background: In the past two decades, regenerative surgeons have focused increasing attention on the potential of gene therapy for treatment of local disorders and injuries. Gene transfer techniques may provide an effective local and short-term induction of growth factors without the limits of other topical therapies. In 2002, Tepper and Mehrara accurately reviewed the topic: given the substantial advancement of research on this issue, an updated review is provided.
Methods: Literature indexed in the National Center for Biotechnology Information database (PubMed) has been reviewed using variable combinations of keywords ("gene therapy," "regenerative medicine," "tissue regeneration," and "gene medicine"). Articles investigating the association between gene therapies and local pathologic conditions have been considered. Attention has been focused on articles published after 2002. Further literature has been obtained by analysis of references listed in reviewed articles.
Results: Gene therapy approaches have been successfully adopted in preclinical models for treatment of a large variety of local diseases affecting almost every type of tissue. Experiences in abnormalities involving skin (e.g., chronic wounds, burn injuries, pathologic scars), bone, cartilage, endothelia, and nerves have been reviewed. In addition, the supporting role of gene therapies to other tissue-engineering approaches has been discussed. Despite initial reports, clinical evidence has been provided only for treatment of diabetic ulcers, rheumatoid arthritis, and osteoarthritis.
Conclusions: Translation of gene therapy strategies into human clinical trials is still a lengthy, difficult, and expensive process. Even so, cutting-edge gene therapy-based strategies in reconstructive procedures could soon set valuable milestones for development of efficient treatments in a growing number of local diseases and injuries.