患者由来iPSCsから視細胞様細胞への分化プロトコル
1Division of Molecular and Cellular Biology, National Institute of Sensory Organs, NHO Tokyo Medical Center, Tokyo 152-8902, Japan.
Abstract:
Retinal biopsies are rarely feasible, limiting direct studies of inherited retinal dystrophies (IRDs). We present a protocol modeling occult macular dystrophy, an autosomal-dominant IRD, using induced pluripotent stem cells (iPSCs) generated from patient blood cells. We describe steps to differentiate iPSCs into photoreceptor-like cells by transducing CRX and NEUROD1 via a tetracycline-inducible PiggyBac vector. We also detail procedures for the validation of photoreceptor markers to assess differentiation. This protocol enables mutation-focused investigation of IRD pathogenesis. For complete details on the use and execution of this protocol, please refer to Pan et al.1.
関連する概念動画
iPS Cell Differentiation
Induced Pluripotent Stem Cells
EPS and iPS Cells in Disease Research


