Liver targeting with rAAV7: balancing tropism with immune profiles

Flotte, Terence R.

dc.contributor.author	Flotte, Terence R.
dc.date	2022-08-11T08:09:59.000
dc.date.accessioned	2022-08-23T16:51:33Z
dc.date.available	2022-08-23T16:51:33Z
dc.date.issued	2021-04-01
dc.date.submitted	2021-07-29
dc.identifier.citation	<p>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. <a href="https://doi.org/10.1038/s41434-021-00230-4">Link to article on publisher's site</a></p>
dc.identifier.issn	0969-7128 (Linking)
dc.identifier.doi	10.1038/s41434-021-00230-4
dc.identifier.pmid	33547423
dc.identifier.uri	http://hdl.handle.net/20.500.14038/41879
dc.description.abstract	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.
dc.language.iso	en_US
dc.relation	<p><a href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&list_uids=33547423&dopt=Abstract">Link to Article in PubMed</a></p>
dc.rights	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 http://creativecommons.org/licenses/by/4.0/.
dc.rights.uri	http://creativecommons.org/licenses/by/4.0/
dc.subject	gene therapy
dc.subject	rAAV7
dc.subject	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
dc.subject	Genetics and Genomics
dc.subject	Therapeutics
dc.title	Liver targeting with rAAV7: balancing tropism with immune profiles
dc.type	Response or Comment
dc.source.journaltitle	Gene therapy
dc.source.volume	28
dc.source.issue	3-4
dc.identifier.legacyfulltext	https://escholarship.umassmed.edu/cgi/viewcontent.cgi?article=5718&context=oapubs&unstamped=1
dc.identifier.legacycoverpage	https://escholarship.umassmed.edu/oapubs/4686
dc.identifier.contextkey	24075774
refterms.dateFOA	2022-08-23T16:51:33Z
html.description.abstract	<p>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.</p>
dc.identifier.submissionpath	oapubs/4686
dc.contributor.department	Department of Pediatrics, Division of Pediatric Pulmonology
dc.source.pages	115-116

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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 http://creativecommons.org/licenses/by/4.0/.

Except where otherwise noted, this item's license is described as 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 http://creativecommons.org/licenses/by/4.0/.