South America is a continent of huge social and economic contrasts, both within and between countries. In this spotlight edition, we present an overview of the continent’s scientific production on gene and cell therapy. We are happy to have active groups from across different countries represented in this issue, including researchers from Brazil, Argentina, Chile, Uruguay, and Colombia. Though some additional groups are working in this area, the work presented here offers a glimpse on research in developing regions, where reagents are often 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 persist as it can be seen by reports showing the evolution of the field of gene therapy in Latin America [1, 2].

As home for about 428,000,000 people, or a little over 5% of the world’s population, South America will be undoubtedly a market for gene and cell therapy products, once they are commercially approved. In that sense, a critical mass is needed to help developing and evaluate these new therapies once they are available. Most groups in South America work closely related to universities, therefore researchers are not only involved in basic and translational investigation. They are also responsible for training a new generation of physicians, veterinarians, pharmacists, and other health related professionals that in the future will be responsible for prescribing and administering gene and cell-based therapeutics. The groups participating in this issue will be among the leaders for this educational challenge and their work shows that they are up to the task.

Several challenges exist for the development of the field in our area and for the applicability of the new treatments. In particular, the elevated cost of the approved treatments will be a burden for the health systems in our region. Moreover, though several leading groups exist in the region, the reduced number of trained professionals and their distribution in the region should be addressed to ensure a widespread availability of game-changing therapies tailored to individual patients.

We are glad our fellow researchers accepted our invitation and managed to submit their work on a variety of topics, ranging from original experimental work, review articles, and comments on the regulatory aspects and history of gene and cell therapy in particular countries. Also, the topics covered range from cancer to inherited diseases, but also for common disorders such as Parkinson and cardiovascular disease. Experimental approaches for gene therapy involve viral and nonviral vectors, gene editing, and DNA vaccines. Cell therapy studies include CAR-T and iPS cells, as well as adipose-derived stem cells.

Finally, we thank Prof. Rafael Yañez-Muñoz for his enthusiastic support for inclusiveness in science. An approach that includes not only encouraging the participation of young investigators but also of people from diverse regional origins. We hope this spotlight edition honors his vision.