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

Heterotopic ossification is the most disabling feature of fibrodysplasia ossificans progressiva, an ultra-rare genetic disorder for which there is currently no prevention or treatment. Most patients with this disease harbor a heterozygous activating mutation (c.617 G > A;p.R206H) in ACVR1. Here,...

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Detalles Bibliográficos
Autores principales: Yang, Yeon-Suk, Kim, Jung-Min, Xie, Jun, Chaugule, Sachin, Lin, Chujiao, Ma, Hong, Hsiao, Edward, Hong, Jaehyoung, Chun, Hyonho, Shore, Eileen M., Kaplan, Frederick S., Gao, Guangping, Shim, Jae-Hyuck
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9579182/
https://www.ncbi.nlm.nih.gov/pubmed/36258013
http://dx.doi.org/10.1038/s41467-022-33956-9

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9579182/
https://www.ncbi.nlm.nih.gov/pubmed/36258013
http://dx.doi.org/10.1038/s41467-022-33956-9

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