Pressing diseases that represent promising targets for gene therapy.

ABSTRACT: 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 f

SUBMITTER: Beitelshees M 

PROVIDER: S-EPMC9890200 | biostudies-literature | 2017 Dec

REPOSITORIES: biostudies-literature