UMMS Affiliation

Department of Pediatrics, Division of Pediatric Pulmonology

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Response or Comment


Congenital, Hereditary, and Neonatal Diseases and Abnormalities | Genetics and Genomics | Therapeutics


The value of intravenous (IV) recombinant adeno-associated virus (rAAV) as a platform for delivery of transgenes to the liver for gene therapy has been well established. The most notable example is the use of a self-complementary rAAV9 vector expressing a high specific activity Factor IX gene in patients with hemophilia B. Since the liver is the primary site for expression of numerous other serum proteins and biochemical pathways, this platform has the potential for very broad utility in a wide variety of genetic diseases. Thus, the optimization of the delivery of rAAV to hepatocytes is of central importance in the field of human gene therapy.


gene therapy, rAAV7

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Copyright © The Author(s) 2021. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit

DOI of Published Version



Flotte TR. Liver targeting with rAAV7: balancing tropism with immune profiles. Gene Ther. 2021 Apr;28(3-4):115-116. doi: 10.1038/s41434-021-00230-4. Epub 2021 Feb 5. PMID: 33547423; PMCID: PMC8087538. Link to article on publisher's site

Journal/Book/Conference Title

Gene therapy

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Creative Commons License

Creative Commons Attribution 4.0 License
This work is licensed under a Creative Commons Attribution 4.0 License.