Department of Pediatrics, Division of Pediatric Pulmonology
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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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
Flotte TR. (2021). Liver targeting with rAAV7: balancing tropism with immune profiles. Open Access Publications by UMMS Authors. https://doi.org/10.1038/s41434-021-00230-4. Retrieved from https://escholarship.umassmed.edu/oapubs/4686
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This work is licensed under a Creative Commons Attribution 4.0 License.