Publication: Gene therapy for achromatopsia
Gene therapy for achromatopsia
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Michalakis, S., Schön, C., Becirovic, E., & Biel, M. (2017). Gene therapy for achromatopsia. Journal of Gene Medicine, 19, e2944–e2944. https://doi.org/10.1002/jgm.2944
Abstract
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The present review summarizes the current status of achromatopsia (ACHM) gene therapy-related research activities and provides an outlook for their clinical application. ACHM is an inherited eye disease characterized by a congenital absence of cone photoreceptor function. As a consequence, ACHM is associated with strongly impaired daylight vision, photophobia, nystagmus and a lack of color discrimination. Currently, six genes have been linked to ACHM. Up to 80% of the patients carry mutations in the genes CNGA3 and CNGB3 encoding the
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Creators (Authors)
- Michalakis, Stylianos
- Schön, Christian
- Biel, Martin
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Citations
Michalakis, S., Schön, C., Becirovic, E., & Biel, M. (2017). Gene therapy for achromatopsia. Journal of Gene Medicine, 19, e2944–e2944. https://doi.org/10.1002/jgm.2944
