Soluble CX3CL1-expressing retinal pigment epithelium cells protect rod photoreceptors in a mouse model of retinitis pigmentosa

CONCLUSIONS: Transgenic hESCs differentiate into hRPE cells and retain sCX3CL1 transgene expression both in vitro and in vivo. Moreover, hRPE cells delivered to the subretinal space of rd10 mice prevented photoreceptor degeneration in a local-acting manner, suggesting that this approach could have applications for preserving photoreceptors in specific subregions of the retina, such as the macula. Overall, our study not only reveals the potential of a combined cell and gene therapy for the treatment of RP, but also the possibility of using hRPE cells to deliver therapeutic biologics in situ to treat diseases over long-term.PMID:37605279 | DOI:10.1186/s13287-023-03434-0

Source: Cell Research - August 22, 2023 Category: Cytology Authors: Eric D Jong Sabiha Hacibekiroglu Lily Guo Evan Sawula Biao Li Chengjin Li Margaret T Ho Molly S Shoichet Valerie A Wallace Andras Nagy Source Type: research