Gene and Cell Therapy

The development of therapeutics to treat human disease has always been a major goal for biomedical research and innovation. Nevertheless, the complexity of the majority of human diseases poses an important and difficult obstacle to overcome. Moreover, the genetic contribution to these conditions complicates the targeting of endogenous and normal processes of cellular functioning, such as transcription and translation. In the past century, the understanding of DNA structure and the development of techniques to manipulate and recombine this molecule allowed the conception of new strategies that could use DNA as a therapeutic agent. In the 1970s, it was proposed for the first time that some human genetic conditions could be treated by the administration of exogenous DNA. The enormous technological advance in the field of biomedicine, with the human genome sequencing or the development of high-throughput techniques, for example, contributed to an effective application of gene therapy in the human context. Recently, the approval of several gene therapy medicines in Europe and the USA definitively established a new paradigm in human disease treatment and opened a new era for gene therapy.