[PDF][PDF] A range of clinical phenotypes associated with mutations in CRX, a photoreceptor transcription-factor gene

MM Sohocki, LS Sullivan, HA Mintz-Hittner… - The American Journal of …, 1998 - cell.com
MM Sohocki, LS Sullivan, HA Mintz-Hittner, D Birch, JR Heckenlively, CL Freund…
The American Journal of Human Genetics, 1998cell.com
Mutations in the retinal-expressed gene CRX (cone-rod homeobox gene) have been
associated with dominant cone-rod dystrophy and with de novo Leber congenital amaurosis.
However, CRX is a transcription factor for several retinal genes, including the opsins and the
gene for interphotoreceptor retinoid binding protein. Because loss of CRX function could
alter the expression of a number of other retinal proteins, we screened for mutations in the
CRX gene in probands with a range of degenerative retinal diseases. Of the 294 unrelated …
Summary
Mutations in the retinal-expressed gene CRX (cone-rod homeobox gene) have been associated with dominant cone-rod dystrophy and with de novo Leber congenital amaurosis. However, CRX is a transcription factor for several retinal genes, including the opsins and the gene for interphotoreceptor retinoid binding protein. Because loss of CRX function could alter the expression of a number of other retinal proteins, we screened for mutations in the CRX gene in probands with a range of degenerative retinal diseases. Of the 294 unrelated individuals screened, we identified four CRX mutations in families with clinical diagnoses of autosomal dominant cone-rod dystrophy, late-onset dominant retinitis pigmentosa, or dominant congenital Leber amaurosis (early-onset retinitis pigmentosa), and we identified four additional benign sequence variants. These findings imply that CRX mutations may be associated with a wide range of clinical phenotypes, including congenital retinal dystrophy (Leber) and progressive diseases such as cone-rod dystrophy or retinitis pigmentosa, with a wide range of onset.
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