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

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

doi:10.1038/s41467-022-33956-9

Nature Communications (Oct 2022)

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

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

Affiliations

Yeon-Suk Yang: Department of Medicine/Division of Rheumatology, UMass Chan Medical School
Jung-Min Kim: Department of Medicine/Division of Rheumatology, UMass Chan Medical School
Jun Xie: Horae Gene Therapy Center, UMass Chan Medical School
Sachin Chaugule: Department of Medicine/Division of Rheumatology, UMass Chan Medical School
Chujiao Lin: Department of Medicine/Division of Rheumatology, UMass Chan Medical School
Hong Ma: Horae Gene Therapy Center, UMass Chan Medical School
Edward Hsiao: Division of Endocrinology and Metabolism, Department of Medicine; the Institute for Human Genetics; the Program in Craniofacial Biology; and the Eli and Edyth Broad Institute of Regeneration Medicine, University of California-San Francisco
Jaehyoung Hong: Department of Mathematical Sciences, Korea Advanced Institute of Science and Technology
Hyonho Chun: Department of Mathematical Sciences, Korea Advanced Institute of Science and Technology
Eileen M. Shore: Department of Orthopaedic Surgery, The Perelman School of Medicine at the University of Pennsylvania
Frederick S. Kaplan: Department of Orthopaedic Surgery, The Perelman School of Medicine at the University of Pennsylvania
Guangping Gao: Horae Gene Therapy Center, UMass Chan Medical School
Jae-Hyuck Shim: Department of Medicine/Division of Rheumatology, UMass Chan Medical School

DOI: https://doi.org/10.1038/s41467-022-33956-9
Journal volume & issue: Vol. 13, no. 1
pp. 1 – 20

Abstract

Read online

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.

Published in Nature Communications

ISSN: 2041-1723 (Online)
Publisher: Nature Portfolio
Country of publisher: United Kingdom
LCC subjects: Science
Website: https://www.nature.com/ncomms/

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