Delivery of adeno-associated virus vectors to the fetal retina: impact of viral capsid proteins on retinal neuronal progenitor transduction.

Surace EM, Auricchio A, Reich SJ, Rex T, Glover E, Pineles S, Tang W, O'Connor E, Lyubarsky A, Savchenko A, Pugh EN, Maguire AM, Wilson JM, Bennett J
J Virol. 2003 77 (14): 7957-63

PMID: 12829835 · PMCID: PMC161923 · DOI:10.1128/jvi.77.14.7957-7963.2003

The development of fetal ocular gene transfer may be useful as a therapeutic tool for the prevention of retinal genetic disorders with congenital or early clinical manifestations. In this study we explored the neural progenitor transduction patterns of adeno-associated virus (AAV) vectors following delivery to the developing retina. Recombinant vectors with the same genome carrying the enhanced green fluorescent protein (EGFP) transgene packaged in capsids of differing serotypes (serotypes 1, 2, and 5, termed AAV2/1, AAV2/2, and AAV2/5, respectively) were created. Delivery of the AAV vectors during early retinal development resulted in efficient and stable transduction of retinal progenitors. Vector surface proteins and the developmental status of the retina profoundly affected viral tropism and transgene distribution. The procedure is not detrimental to retinal development and function and therefore provides a safe delivery vehicle for potential therapeutic applications and a means of assessing the mechanisms of retina development and disease.

MeSH Terms (19)

Animals Capsid Proteins Cell Differentiation Dependovirus Female Genetic Therapy Genetic Vectors Gene Transfer Techniques Green Fluorescent Proteins Luminescent Proteins Mice Mice, Inbred C57BL Neurons Pigment Epithelium of Eye Pregnancy Retina Stem Cells Transduction, Genetic Transgenes

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