- Louie, Carrie M;
- Caridi, Gianluca;
- Lopes, Vanda S;
- Brancati, Francesco;
- Kispert, Andreas;
- Lancaster, Madeline A;
- Schlossman, Andrew M;
- Otto, Edgar A;
- Leitges, Michael;
- Gröne, Hermann-Josef;
- Lopez, Irma;
- Gudiseva, Harini V;
- O'Toole, John F;
- Vallespin, Elena;
- Ayyagari, Radha;
- Ayuso, Carmen;
- Cremers, Frans PM;
- den Hollander, Anneke I;
- Koenekoop, Robert K;
- Dallapiccola, Bruno;
- Ghiggeri, Gian Marco;
- Hildebrandt, Friedhelm;
- Valente, Enza Maria;
- Williams, David S;
- Gleeson, Joseph G
Degeneration of photoreceptors is a common feature of ciliopathies, owing to the importance of the specialized ciliary structure of these cells. Mutations in AHI1, which encodes a cilium-localized protein, have been shown to cause a form of Joubert syndrome that is highly penetrant for retinal degeneration. We show that Ahi1-null mice fail to form retinal outer segments and have abnormal distribution of opsin throughout their photoreceptors. Apoptotic cell death of photoreceptors occurs rapidly between 2 and 4 weeks of age in these mice and is significantly (P = 0.00175 and 0.00613) delayed by a reduced dosage of opsin. This phenotype also shows dosage-sensitive genetic interactions with Nphp1, another ciliopathy-related gene. Although it is not a primary cause of retinal blindness in humans, we show that an allele of AHI1 is associated with a more than sevenfold increase in relative risk of retinal degeneration within a cohort of individuals with the hereditary kidney disease nephronophthisis. Our data support context-specific roles for AHI1 as a contributor to retinopathy and show that AHI1 may explain a proportion of the variability in retinal phenotypes observed in nephronophthisis.