AAV-Mediated Targeting of the Activin A-ACVR1<sup>R206H</sup> Signaling in Fibrodysplasia Ossificans Progressiva

AAV-Mediated Targeting of the Activin A-ACVR1<sup>R206H</sup> Signaling in Fibrodysplasia Ossificans Progressiva

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized by progressive disabling heterotopic ossification (HO) at extra-skeletal sites. Here, we developed adeno-associated virus (AAV)-based gene therapy that suppresses trauma-induced HO in FOP mice harboring a het...

Full description

Bibliographic Details
Main Authors: Yeon-Suk Yang, Chujiao Lin, Hong Ma, Jun Xie, Frederick S. Kaplan, Guangping Gao, Jae-Hyuck Shim
Format: Article
Language:English
Published: MDPI AG 2023-09-01
Series:Biomolecules
Subjects:
fibrodysplasia ossificans progressiva
heterotopic ossificans
Activin A
ACVR1
AAV
gene therapy
Online Access:https://www.mdpi.com/2218-273X/13/9/1364

Internet

https://www.mdpi.com/2218-273X/13/9/1364

Similar Items