Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here

UMMS Affiliation

Horae Gene Therapy Center; Department of Pediatrics

Publication Date


Document Type



Clinical Trials | Genetic Phenomena | Genetics and Genomics | Therapeutics | Virology


The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies in Europe and the United States are landmark achievements in the history of modern science. These approvals are also anticipated to herald the emergence of a new class of therapies for monogenic disorders, which had hitherto been considered untreatable. These events can be viewed as stemming from the convergence of several important historical trends: the study of basic virology, the development of genomic technologies, the imperative for translational impact of National Institutes of Health-funded research, and the development of economic models for commercialization of rare disease therapies. In this review, these historical trends are described and the key developments that have enabled clinical rAAV gene therapies are discussed, along with an overview of the current state of the field and future directions.


adeno-associated virus, AAV, gene therapy, genetic disease, genome editing, clinical trial

DOI of Published Version



Annu Rev Virol. 2019 Sep 29;6(1):601-621. doi: 10.1146/annurev-virology-092818-015530. Epub 2019 Jul 5. Link to article on publisher's site

Journal/Book/Conference Title

Annual review of virology

Related Resources

Link to Article in PubMed

PubMed ID