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pro vyhledávání: '"Janell L. M. Smith"'
Autor:
Francesco Limone, Jana M. Mitchell, Irune Guerra San Juan, Janell L. M. Smith, Kavya Raghunathan, Alexander Couto, Sulagna Dia Ghosh, Daniel Meyer, Curtis J. Mello, James Nemesh, Brittany M. Smith, Steven McCarroll, Olli Pietiläinen, Ralda Nehme, Kevin Eggan
SUMMARYHuman pluripotent stem cells (hPSCs) are a powerful tool for disease modelling and drug discovery, especially when access to primary tissue is limited, such as in the brain. Current neuronal differentiation approaches use either small molecule
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::6ab4d3097d8c0e9b075a3eaa7ced5c80
https://doi.org/10.1101/2022.01.12.476020
https://doi.org/10.1101/2022.01.12.476020
Autor:
Jordan Blondelle, Ilya Gertsman, Celine J. Rocca, Janell L. M. Smith, Joseph H. Haquang, Spencer Goodman, Charles J. Heyser, Jennifer N. Dulin, Stephanie Cherqui
Publikováno v:
Science translational medicine, vol 9, iss 413
Friedreich’s ataxia (FRDA) is an incurable autosomal recessive neurodegenerative disease caused by reduced expression of the mitochondrial protein frataxin due to an intronic GAA-repeat expansion in the FXN gene. We report the therapeutic efficacy