基于药物再利用的联合治疗在临床前视网膜病变模型中证明了与突变无关的功效。
Mesh : Animals Drug Repositioning Mice Disease Models, Animal Dogs Retinitis Pigmentosa / drug therapy genetics Mutation Cyclic Nucleotide Phosphodiesterases, Type 6 / genetics metabolism Receptors, G-Protein-Coupled / genetics metabolism Mice, Knockout Leber Congenital Amaurosis / drug therapy genetics Bromocriptine / pharmacology therapeutic use cis-trans-Isomerases / genetics metabolism Humans Drug Therapy, Combination Mice, Inbred C57BL Retinal Cone Photoreceptor Cells / drug effects metabolism pathology Female Cyclic AMP / metabolism Retinal Degeneration / drug therapy genetics Male Calcium / metabolism
来 源:
DOI:10.1038/s41467-024-50033-5
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