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C. Punzo, C. Cepko; Ultrasound Guided in Utero Gene Delivery: A Tool to Manipulate Early Born Retinal Cell Types. Invest. Ophthalmol. Vis. Sci. 2007;48(13):4603.
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Retinal degeneration (RD) is a disease involving loss of photoreceptors. New approaches to understand the progression of the disease, such as micro-array analysis, have yielded many potential genes involved in the pathology. To rapidly test a large number of genes that may influence RD, a method was established to stably transfer genes of interest into a diseased retina.
Ultra sound guided in utero gene transfer is used to deliver the gene of interest either by transfection (electroporation) or via infection (virus for stable integration) into the retina. Cell specificity is achieved by introducing cell type specific promoters.
Viral infections beginning at embryonic day 9.5 lead to stable integration and expression through development and adulthood. Electroporation beginning at embryonic day 13.5 lead to transient gene expression.
Ultrasound-guided in utero gene transfer offers a number of potential advantages in understanding retinal degeneration. This effective tool has allowed for easy and successful targeting of all retinal cell types at specific stages of development and as a result, a fast prescreening of gene function in the retina.
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