One More Controversy: Adeno-Associated Virus in Stem Cells
Department of Pediatrics, Division of Pulmonology
Genetics and Genomics | Therapeutics
Among the earliest treatment paradigms envisioned for gene therapy was the ex vivo genetic modification of autologous stem cells, particularly in the context of bone-marrow transplantation. Many early applications of gammaretrovirus and lentivirus vectors have used this platform, with the primary focus being on hematopoietic stem cells (HSCs). In the April 2017 special issue of Human Gene Therapy, a number of crucial issues in the field of adeno-associated virus (AAV) gene therapy were discussed. However, the issue of the utility of recombinant AAV (rAAV) gene therapy for stem-cell transduction was not directly addressed in that issue.
The problem is now grappled with directly in this June issue of Human Gene Therapy.
DOI of Published Version
Hum Gene Ther. 2017 Jun;28(6):449. doi: 10.1089/hum.2017.29043.trf. Link to article on publisher's site
Human gene therapy
Flotte, Terence R., "One More Controversy: Adeno-Associated Virus in Stem Cells" (2017). Pediatric Publications and Presentations. 156.