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

YS Yang, JM Kim, J Xie, S Chaugule, C Lin… - Nature …, 2022 - nature.com
Heterotopic ossification is the most disabling feature of fibrodysplasia ossificans
progressiva, an ultra-rare genetic disorder for which there is currently no prevention or …

[HTML][HTML] AAV-Mediated Targeting of the Activin A-ACVR1R206H Signaling in Fibrodysplasia Ossificans Progressiva

YS Yang, C Lin, H Ma, J Xie, FS Kaplan, G Gao… - Biomolecules, 2023 - mdpi.com
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized
by progressive disabling heterotopic ossification (HO) at extra-skeletal sites. Here, we …

An Acvr1 R206H knock‐in mouse has fibrodysplasia ossificans progressiva

SA Chakkalakal, D Zhang, AL Culbert… - Journal of bone and …, 2012 - academic.oup.com
Fibrodysplasia ossificans progressiva (FOP; MIM# 135100) is a debilitating genetic disorder
of dysregulated cellular differentiation characterized by malformation of the great toes during …

[HTML][HTML] An anti-ACVR1 antibody exacerbates heterotopic ossification by fibro-adipogenic progenitors in fibrodysplasia ossificans progressiva mice

JB Lees-Shepard, SJ Stoessel… - The Journal of …, 2022 - Am Soc Clin Investig
Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disease characterized by
progressive and catastrophic heterotopic ossification (HO) of skeletal muscle and associated …

[HTML][HTML] Activin-dependent signaling in fibro/adipogenic progenitors causes fibrodysplasia ossificans progressiva

JB Lees-Shepard, M Yamamoto, AA Biswas… - Nature …, 2018 - nature.com
Fibrodysplasia ossificans progressiva (FOP) is a rare autosomal-dominant disorder
characterized by progressive and profoundly disabling heterotopic ossification (HO). Here …

Two tissue-resident progenitor lineages drive distinct phenotypes of heterotopic ossification

D Dey, J Bagarova, SJ Hatsell, KA Armstrong… - Science translational …, 2016 - science.org
Fibrodysplasia ossificans progressiva (FOP), a congenital heterotopic ossification (HO)
syndrome caused by gain-of-function mutations of bone morphogenetic protein (BMP) type I …

ACVR1 p. Q207E causes classic fibrodysplasia ossificans progressiva and is functionally distinct from the engineered constitutively active ACVR1 p. Q207D variant

J Haupt, A Deichsel, K Stange, C Ast… - Human molecular …, 2014 - academic.oup.com
Fibrodysplasia ossificans progressiva (FOP) is a disabling genetic disorder of progressive
heterotopic ossification (HO). Here, we report a patient with an ultra-rare point mutation [c …

Depletion of Mast Cells and Macrophages Impairs Heterotopic Ossification in an Acvr1R206H Mouse Model of Fibrodysplasia Ossificans Progressiva

MR Convente, SA Chakkalakal, EJ Yang… - Journal of Bone and …, 2018 - academic.oup.com
Heterotopic ossification (HO) is a clinical condition that often reduces mobility and
diminishes quality of life for affected individuals. The most severe form of progressive HO …

Restoration of normal BMP signaling levels and osteogenic differentiation in FOP mesenchymal progenitor cells by mutant allele-specific targeting

J Kaplan, FS Kaplan, EM Shore - Gene therapy, 2012 - nature.com
Fibrodysplasia ossificans progressiva (FOP) is a rare autosomal dominant disorder of
progressive heterotopic ossification for which there is presently no cure. FOP is caused by a …

Neofunction of ACVR1 in fibrodysplasia ossificans progressiva

K Hino, M Ikeya, K Horigome… - Proceedings of the …, 2015 - National Acad Sciences
Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disease characterized by
extraskeletal bone formation through endochondral ossification. FOP patients harbor point …