May 2007
Volume 48, Issue 13
ARVO Annual Meeting Abstract  |   May 2007
Ultrasound Guided in Utero Gene Delivery: A Tool to Manipulate Early Born Retinal Cell Types
Author Affiliations & Notes
  • C. Punzo
    Genetics, Harvard Medical School, Boston, Massachusetts
  • C. Cepko
    Genetics, Harvard Medical School, Boston, Massachusetts
  • Footnotes
    Commercial Relationships C. Punzo, None; C. Cepko, None.
  • Footnotes
    Support NIH Grant EY014466, EMBO
Investigative Ophthalmology & Visual Science May 2007, Vol.48, 4603. doi:
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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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      © ARVO (1962-2015); The Authors (2016-present)

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Purpose:: 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.

Methods:: 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.

Results:: 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.

Conclusions:: 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.

Keywords: gene transfer/gene therapy • retinal degenerations: cell biology • photoreceptors 

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