Pressing diseases that represent promising targets for gene therapy

Over time, there has been a growing interest in the application of gene therapy within the healthcare industry as demonstrated by the nearly 3,000 clinical trials associated with gene therapy that are listed in clinicaltrials.gov. However, there are various difficulties associated with gene therapy that have limited the realization of licensed gene therapies to only a handful of treatments. Furthermore, efforts to develop gene therapeutics have been narrowly focused and most clinical trials have sought to develop treatments for cancer (64.6%), monogenic diseases (10.5%), infectious diseases (7.4%), and cardiovascular diseases (7.4%). In addition, nearly 70% of clinical trials have utilized viral-based delivery systems, despite various concerns associated with this strategy. Each of these factors highlights the lack of diversity in the development of gene therapeutics that should be addressed. In recent years, developments in gene manipulation and delivery such as CRISPR and non-viral vectors (e.g., liposomes) demonstrate promise for improving outcomes for gene therapy. The increased fidelity and capacity afforded by these technologies provide the potential to improve upon contemporary gene therapy approaches and enable the development of treatments for less-emphasized disorders. In this review, we provide a summary of gene delivery technology and discuss various developments in gene therapy technology. We conclude by proposing several genetic conditions that represent promising targets for gene therapy given recent developments in gene delivery and manipulation.