Adeno-associated viral vectors based on serotype 3b use components of the fibroblast growth factor receptor signaling complex for efficient transduction.
Journal Article (Journal Article)
Adeno-associated virus type 3b (AAV3b) has been largely ignored by gene therapists because of the inability of vectors based on this serotype to transduce target tissues efficiently. Here we describe a phenomenon unique to AAV3b in that vectors based on this serotype mediate enhanced transduction in the presence of heparin. Among the many biological functions attributed to heparin, its interaction with, and ability to regulate, several growth factors (GFs) and growth factor receptors (GFRs) has been well characterized. Using GFR-overexpressing cell lines, soluble GFs and heparins, as well as specific GFR inhibitors, we have demonstrated a requirement for fibroblast growth factor receptor-2 (FGFR2) and FGF1 in the heparin-mediated augmentation of AAV3b vector transduction. In contrast to AAV2, we establish that heparin can be used as an adjunct with AAV3b to further increase transduction in a variety of cells and target tissues, additionally suggesting that AAV3b may be an attractive viral vector for clinical use during procedures in which heparin is used. In summary, AAV3b exhibits FGFR2-dependent, markedly enhanced transduction efficiency in the presence of heparin and FGFs, which could make it a useful vector for gene therapy in a variety of human diseases.
Full Text
Duke Authors
Cited Authors
- Messina, EL; Nienaber, J; Daneshmand, M; Villamizar, N; Samulski, J; Milano, C; Bowles, DE
Published Date
- October 2012
Published In
Volume / Issue
- 23 / 10
Start / End Page
- 1031 - 1042
PubMed ID
- 22680698
Pubmed Central ID
- PMC3472518
Electronic International Standard Serial Number (EISSN)
- 1557-7422
Digital Object Identifier (DOI)
- 10.1089/hum.2012.066
Language
- eng
Conference Location
- United States