A snapshot of gene therapy in Latin America

Abstract

Gene therapy attempts the insertion and expression of exogenous genetic material in cells for therapeutic purposes. Conceived in the 1960s, gene therapy reached its first clinical trial at the end of the 1980s and by December 2013 around 600 genuine open clinical trials of gene therapy were registered at NIH Clinical Trials Database. Here, we summarize the current efforts towards the development of gene therapy in Latin America. Our survey shows that the number of scientists involved in the development of gene therapy and DNA vaccines in Latin America is still very low. Higher levels of investment in this technology are necessary to boost the advancement of innovation and intellectual property in this field in a way that would ease both the social and financial burden of various medical conditions in Latin America.

RNAi-based Gene Therapy for Blood Genetic Diseases

“…Insertion and expression of specific exogenous genetic materials via the transfer of nucleic acids directly in vivo, or through modified cells in vitro, correct a cellular dysfunction or provide a new cellular function. It has the potential to cure any genetic disease with long-lasting therapeutic benefits [1,2]. Gene therapy can be classified into (i) germ line and (ii) somatic line gene therapy types.…”

“…According to the mechanisms, gene therapy may include three major categories: (a) direct modulation of the disease-causing gene, which can be applied to monogenic hereditary diseases; (b) indirect treatment through gene modulation, which can be applied to multifactorial diseases; and (c) immunotherapy by gene modulation (DNA vaccines), which leads to the synthesis of the relevant antigen or an adjuvant [1].…”

Spotlight edition on South America

“…Though more groups are working in this area, the work presented in this Spotlight issue offers a glimpse of research in developing regions, where reagents are mostly imported (and therefore expensive and not readily available) and financial support for scientific activities is often scarce and inconsistent. In spite of that, many groups continue researching in this area, as can be seen in reports describing the evolution of the field of gene therapy in Latin America [1,2].…”