Friedrich Miescher Institute for Biomedical Research

Novartis Forschungsstiftung Zweigniederlassung Friedrich Miescher Institute for Biomedical Research

University Hospital Basel

Jacek Krol

Botond Roska

Josephine Juettner

Witold Filipowicz

Markus Heim

Magdalena Filipowicz

Ilona Markiewicz

The present invention to a method for expressing an exogenous gene specifically in cells of the retinal pigment epithelium of a primate, this method comprising the step of delivering an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1, or consisting of a nucleic acid sequence of at least 400 bp having at least 80% overall identity to the sequence of SEQ ID NO:1, to cells of the retinal pigment epithelium of the primate, wherein this isolated nucleic acid molecule specifically leads to the expression of an exogenous gene in cells of the retinal pigment epithelium of primates when a nucleic acid sequence coding for the exogenous gene is operatively linked to this isolated nucleic acid molecule.

Primate retinal pigment epithelium cell-specific promoter

The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 1000 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in cells of the retinal pigment epithelium of a gene when operatively linked to a nucleic acid sequence coding for said gene.

SynPIII, a promoter for the specific expression of genes in retinal pigment epithelium

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Basel, Switzerland

Jacek Krol