Zobrazeno 1 - 3
of 3
pro vyhledávání: '"Rain Dimen"'
Autor:
Rena Baek, Kimberly Coughlan, Lei Jiang, Min Liang, Lei Ci, Harkewal Singh, Hannah Zhang, Neeraj Kaushal, Ivana Liric Rajlic, Linh Van, Rain Dimen, Alexander Cavedon, Ling Yin, Lisa Rice, Andrea Frassetto, Lin Guey, Patrick Finn, Paolo G. V. Martini
Publikováno v:
Nature Communications, Vol 15, Iss 1, Pp 1-13 (2024)
Abstract Messenger RNA (mRNA) therapeutics delivered via lipid nanoparticles hold the potential to treat metabolic diseases caused by protein deficiency, including propionic acidemia (PA), methylmalonic acidemia (MMA), and phenylketonuria (PKU). Here
Externí odkaz:
https://doaj.org/article/fc0132ec8a774c5fbcc5732a7b7a7a0e
Autor:
Bijina Balakrishnan, Xinhua Yan, Marshall D. McCue, Olivia Bellagamba, Aaron Guo, Felicity Winkler, Jason Thall, Lisa Crawford, Rain Dimen, Sara Chen, Sean McEnaney, Yiman Wu, Mike Zimmer, Joe Sarkis, Paolo G.V. Martini, Patrick F. Finn, Kent Lai
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 1, Pp 101191- (2024)
Despite the implementation of lifesaving newborn screening programs and a galactose-restricted diet, many patients with classic galactosemia develop long-term debilitating neurological deficits and primary ovarian insufficiency. Previously, we showed
Externí odkaz:
https://doaj.org/article/d2fda7725fe9434a86e2d785a3e6d368
Publikováno v:
Clinical Trials Week; 5/27/2024, p900-900, 1p