Targeting molecular pathways for the treatment of inherited retinal degeneration

• 作 者: Meltem Kutluer Li Huang Valeri
• 机 构: DepartmentofLifeSciences
• 出 处: 中国神经再生研究:英文版
• 发表日期: 2020年10期
• 关键词: ACHROMATOPSIA calcium CALPAINS CALPASTATIN CONGENITAL stationary night blindness
• 基金项目: This work was supported by grants from the Telethon Foundation(GGP14180,GGP19113),the European Union(LSHGCT-2005-512036 and transMed,MSCA-ITN-2017-765441)(all to VM).

Inherited retinal degeneration is a major cause of incurable blindness characterized by loss of retinal photoreceptor cells.Inherited retinal degeneration is characterized by high genetic and phenotypic heterogeneity with several genes mutated in patients