PNAS: Researchers have found drugs for retinal malnutrition

December 3, 2020 /--- In a recent study, a team of researchers at LSU Health in New Orleans reported for the first time that in mouse models, one of the inhibitors of the RPE65 gene was removed to prevent the degradation of cone cells caused by genetic mutations.
their findings were published in PNAS.
reported that more than 100 DNA variants in the RPE65 gene are disease-causing mutations that cause retinal degenerative disease.
include a genetic childhood blinding disorder called LCA.
although a new gene therapy can improve vision in some people with RPE65 gene mutations, there is currently no effective treatment to prevent retinal degeneration in LCA.
(Photo Source: www.pixabay.com) before researchers identified three RPE65 inhibitors.
a type of fatty acid transporter protein 4 (FATP4) involved in this study.
found that in LCA mouse models lacking FATP4 in the retina, the survival rate of cone-sensing cells increased nearly tenfold.
also found that reducing the fatP4 portion of the retina improved the survival and visual function of cone cells.
the findings identified FATP4 as a promising therapeutic target for preserving the patient's daytime color perception.
"FATP4's role in the progress of retinal malnutrition associated with the RPE65 mutation is completely unknown," said Dr. Nicolas Bazan.
Jin Minghao, professor of neuroscience and ophthalmological sciences at LSU Health New, added: "Our findings allow us to envision strategies to reduce the degradation of cone cells and vision loss in patients with RPE65 mutations and other mutations.
" (Bioon.com) Source: Researchers discovery drug development target for retinal dystrophies Original source: Songhua Li et al, Inverse correlation between fatty acid transport protein 4 and vision in Leber congenital amaurosis with RPE65, Proceedings of the National Academy of Sciences (2020). DOI: 10.1073/pnas.2012623117。