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Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery

Authors :
Yeon-Suk Yang
Jung-Min Kim
Jun Xie
Sachin Chaugule
Chujiao Lin
Hong Ma
Edward Hsiao
Jaehyoung Hong
Hyonho Chun
Eileen M. Shore
Frederick S. Kaplan
Guangping Gao
Jae-Hyuck Shim
Source :
Nature Communications, Vol 13, Iss 1, Pp 1-20 (2022)
Publication Year :
2022
Publisher :
Nature Portfolio, 2022.

Abstract

Fibrodysplasia ossificans progressiva is an ultra-rare genetic disorder with progressive heterotopic ossification. Yang et al develop different gene therapy approaches and show their efficacy in mouse models and in human induced pluripotent stem cells.

Subjects

Subjects :
Science

Details

Language :
English
ISSN :
20411723
Volume :
13
Issue :
1
Database :
Directory of Open Access Journals
Journal :
Nature Communications
Publication Type :
Academic Journal
Accession number :
edsdoj.261d337857a4449b8131eb048cff41f6
Document Type :
article
Full Text :
https://doi.org/10.1038/s41467-022-33956-9