Articles

This paper leverages gene and cell therapy research in diverse disorders ranging from monogenic to infectious diseases to cancer and emerging breakthroughs, where one can harness individual genes or a synthetic gene sequence designed based on a shared molecular pattern in infected cells to better fight various disorders [1]. A pivotal task is to predict the performances of candidate gene therapies to guide clinical translational research using methods such as retrospective bioinformatic analyses. Implementing them to a large-scale gene therapy database reveals that it is feasible to construct and apply well-performing interpretable, supervised learning models [2]. Preliminary evidence of machine learning approaches' statistical significance helps clinicians and biomedical researchers, market participants, and regulatory and economic experts derive relevant, practical applications, thereby enhancing the deployment of gene therapy and genomics to achieve positive, long-term growth for humanity while alleviating the ongoing worldwide economic burden precipitated by prolonged and recurring diseases. Deploying machine learning techniques to accelerate gene and cell therapy drug development and trials shall also mitigate the existing obstacle of limited patient access to emerging, transformative medical innovations such as gene therapy due to skyrocketing prices, which often herald gene therapy products as the world's most expensive medicines [3]. Moreover, in preventing patients from accessing effective, life-saving genetic medicines, there commonly exists a multidimensional access gap encompassing the availability, affordability, and quality or acceptability of these clinical treatments. The ensuing substantial gap has repeatedly been documented and mainly emanates from differential institutional and socio-political choices around resource allocation at international and domestic levels [4]. Particularly, it is also due to the stringent licensure and regulatory approval processes underpinned by insufficient evidence for novel safety and clinical efficacy profiles for genetic therapies in multiple micro-local diagnoses and subpopulations. We believe that a higher likelihood of gene therapy adoption shall result when the clinical evidence path contains adequate representation from the most diverse and relevant patient populations [5].

Neurodegeneration and aging are pressing issues with significant personal, economic, ethical, and social consequences. However, the underlying biological mechanisms of these conditions remain largely unknown, making the development of effective treatments challenging. The difficulty in early detection and diagnosis of neurodegenerative diseases further compounds the issue. Recent advancements in genetics, genomics, and brain imaging technology hold great promise for improving our understanding of neurodegeneration and aging, as well as the development of personalized medicine and new drugs and therapies. Addressing these challenges will require a multi-disciplinary and collaborative approach from researchers in various fields. This Special Issue offers valuable insights and perspectives on this critical area of research, which can help advance our understanding and improve the health and well-being of our aging population.