- Nociari, Marcelo M;
- Lehmann, Guillermo L;
- Bay, Andres E Perez;
- Radu, Roxana A;
- Jiang, Zhichun;
- Goicochea, Shelby;
- Schreiner, Ryan;
- Warren, J David;
- Shan, Jufang;
- de Beaumais, Ségolène Adam;
- Ménand, Mickaël;
- Sollogoub, Matthieu;
- Maxfield, Frederick R;
- Rodriguez-Boulan, Enrique
Accumulation of lipofuscin bisretinoids (LBs) in the retinal pigment epithelium (RPE) is the alleged cause of retinal degeneration in genetic blinding diseases (e.g., Stargardt) and a possible etiological agent for age-related macular degeneration. Currently, there are no approved treatments for these diseases; hence, agents that efficiently remove LBs from RPE would be valuable therapeutic candidates. Here, we show that beta cyclodextrins (β-CDs) bind LBs and protect them against oxidation. Computer modeling and biochemical data are consistent with the encapsulation of the retinoid arms of LBs within the hydrophobic cavity of β-CD. Importantly, β-CD treatment reduced by 73% and 48% the LB content of RPE cell cultures and of eyecups obtained from Abca4-Rdh8 double knock-out (DKO) mice, respectively. Furthermore, intravitreal administration of β-CDs reduced significantly the content of bisretinoids in the RPE of DKO animals. Thus, our results demonstrate the effectiveness of β-CDs to complex and remove LB deposits from RPE cells and provide crucial data to develop novel prophylactic approaches for retinal disorders elicited by LBs.