Development and evaluation of siRNA-mediated gene silencing strategies for ADO2 therapy utilizing iPSCs model and DMPC-SPIONs delivery system

Abstract Background Autosomal dominant osteodystrophy type II (ADO2) is an inherited disease characterized by an abnormal increase in bone mineral density, and CLCN7 (R286W) is its most common causative mutation. The aim of this study was to explore the new idea of siRNA technology applied to the in...

Full description

Saved in:
Bibliographic Details
Main Authors: Jiajun Xu, Gengshuo Chen, Chune Mo, Yu Sha, Sha Luo, Minglin Ou
Format: Article
Language:English
Published: BMC 2025-02-01
Series:Stem Cell Research & Therapy
Subjects:
Online Access:https://doi.org/10.1186/s13287-025-04151-6
Tags: Add Tag
No Tags, Be the first to tag this record!