Ubiquitination-targeted therapies improve BMD iPSC myogenic cell engraftment and dystrophin expression in vivo
Year: 2025
Type: article
Abstract: engraftment demonstrated significant improvement in BMD iPSC myogenic cell survival and dystrophin expression in the animals treated with two molecules: a TRIM63 inhibitor and an α-synuclein aggregation inhibitor. These findings provide promising evidence for the potential therapeutic efficacy of these ubiquitination pathway inhibitors to improve muscle progenitor cell survival and dystrophin expression in BMD patients.
Authors: Muchen Liu, Somik Chatterjee, Jianbo Wu, Zeinab Kashaniasl, Ashok Kumar, Chunru Lin, Radbod Darabi
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $3,900
Awards: G562567308, G8762679033
OpenAlex ID: W4411334384